Beverly L. Davidson, PhD

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Beverly L. Davidson, PhD, is Director of the Raymond G. Perelman Center for Cellular and Molecular Therapeutics and Chief Scientific Strategy Officer at Children’s Hospital of Philadelphia.

Areas of Expertise: Neurodegenerative disease, RNA biology, Gene therapy, Animal models
Appointments and Referrals: 1-800-TRY-CHOP (1-800-879-2467)


Dr. Beverly Davidson's lab is focused on genetic diseases that affect the brain, studying the mechanisms by which mutant gene products contribute to disease, and why certain brain regions are more susceptible. The team employs advanced molecular methods, sequencing and imaging modalities in animal models. The lab is also engaged in the development of next generation therapeutics for inherited disorders, including the engineering of novel gene therapy vector capsids and cargo to approach tissue and cell type specific treatments.

Education and Training


Postdoctoral Fellow - University of Michigan, Ann Arbor, MI
Research Investigator - University of Michigan, Ann Arbor, MI

Graduate Degree

PhD in Biological Chemistry - University of Michigan, Ann Arbor, MI

Titles and Academic Titles

Chief Scientific Strategy Officer

Director, Raymond G. Perelman Center for Cellular and Molecular Therapeutics

Director, Research Vector Core

Arthur V. Meigs Chair in Pediatrics at Children’s Hospital of Philadelphia

Professor of Pathology and Laboratory Medicine, Perelman School of Medicine at the University of Pennsylvania.

Departments and Services

Research Interests

Inherited genetic diseases that cause central nervous system dysfunction
Recessive, childhood onset neurodegenerative disease
Lysosomal storage diseases
Battens disease
Dominant genetic diseases
CAG repeat disorders
Huntington’s disease
Spinal cerebellar ataxia
Understanding how noncoding RNAs participate in neural development and neurodegenerative diseases processes




Simpson, BP, Yrigollen CM, Izea A, Davidson BL: Targeted long-read sequencing captures CRISPR editing and AAV integration outcomes in brain. Mol Ther S1525-0016(23)00004-7, Jan 2023 Notes: DOI: 10.1016/j.ymthe.2023.01.004.

Ranum PT, Tecedor L, Keiser MS, Chen YH, Leib DE, Liu X, Davidson, BL: Cochlear transduction via cerebrospinal fluid delivery of AAV in non-human primates. Mol Ther S1525-0016(22)00754-7, Jan 2023 Notes: doi: 10.1016/j.ymthe.2022.12.018.


Fang L, Monteys AM, Durr A, Keiser M, Cheng C, Harapanahalli A, Gonzalez-Alegre P, Davidson BL, Wang K: Haplotyping SNPs for allele-specific gene editing of the expanded huntingtin allele using long-read sequencing. HGG Adv 4(1): 100146, Sept 2022 Notes: DOI: 10.1016/j.xhgg.2022.100146.

St-Cyr S, Child DD, Giaime E, Smith AR, Pascua CJ, Hahm S, Saiah E, Davidson BL: Huntington's disease phenotypes are improved via mTORC1 modulation by small molecule therapy. PLoS One 17(8): e0273710, August 2022 Notes: doi: 10.1371/journal.pone.0273710.

Sabatino DE, Bushman FD, Chandler RJ, Crystal RG, Davidson BL, Dolmetsch R, Eggan KC, Gao G, Gil-Farina I, Kay MA, McCarty DM, Montini E, Ndu A, Yuan J (American Society of Gene and Cell Therapy (ASGCT) Working Group on AAV Integration): Evaluating the state of the science for adeno-associated virus integration: An integrated perspective. Mol Ther 30(8): 2646-2663, Aug 2022.

Dawicki-McKenna JM, Felix AJ, Waxman EA, Cheng C, Amado DA, Ranum PT, Bogush A, Dungan LV, Heller EA, French DL, Davidson BL, Prosser BL: Mapping PTBP splicing in human brain identifies targets for therapeutic splice switching SNYGAP1. bioRxiv July 2022 Notes: doi:

Davidson BL, Gao G, Berry-Kravis E, Bradbury AM, Bonnemann C, Buxbaum JD, Corcoran G, Gray SJ, Gray-Edwards H, Kleiman RJ, Shaywitz AJ, Wang D, Zoghbi HY, Flotte TR, Tauscher-Wisniewski S, Tifft CF, Sahin M, (Gene Therapy Workshop Faculty): Gene-based therapeutics for rare genetic neurodevelopmental psychiatric disorders. Mol Ther 30(7): 2416-2428, July 2022.

Sabatino DE,, Bushman FD (Chair), Chandler RJ, Crystal RG, Davidson BL, Dolmetsch R, Eggan KC, Gao G, Gil-Farina I, Kay MA, McCarty DM, Montini E, Ndu A, Yuan J: Evaluating the State of the Science for Adeno-Associated Virus (AAV) Integration:An Integrated Perspective. Mol Ther S1525-0016(22)00364-1, June 2022 Notes: doi: 10.1016/j.ymthe.2022.06.004.

Davidson BL, Gao G, Berry-Kravis E, Bradbury A, Bonnemann C, Buxbaum JD, Corcoran GR, Gray SJ, Gray-Edwards H, Kleiman RJ, Shaywitz A, Wang D, Zoghbi HY, Flotte TR, Tauscher-Wisniewski S, Tifft CF, Sahin M; Gene Therapy Workship Faculty: Gene-based therapeutics for rare genetic neurodevelopmental psychiatric disorders. Mol Ther Page: S1525-0016(22)00312-4, May 2022.

Yrigollen CM, Simpson B, Lim E, Chen YH, Davidson B: The molecular and behavioral phenotypes altered in CGG knock-in mice edited using CRISPR. Presented at the 25th annual meeting of the American Society for Gene and Cell Therapy, Washington, DC Page: Poster M-160, May 2022.


Victor MB, Richner M, Olsen HE, Lee SW, Monteys AM, Ma C, Huh CJ, Zhang B, Davidson BL, Yang XW, Yoo AS. Striatal neurons directly converted from Huntington's disease patient fibroblasts recapitulate age-associated disease phenotypes. Nat Neurosci. 2018 Mar;21(3):341-352.

Chen Y, Zheng S, Tecedor L, Davidson BL. Overcoming limitations inherent in sulfamidase to improve mucopolysaccharidosis IIIA gene therapy. Mol Ther. 2018 Apr 4;26(4):1118-1126.

Schultz ML, Tecedor L, Lysenko E, Ramachandran S, Stein CS, Davidson BL. Modulating membrane fluidity    corrects Batten disease phenotypes in vitro and in vivo. Neurobiol Dis. 115:182-193, 2018.  doi:  10.1016/j.nbd.2018.04.010. [Epub ahead of print]

Child DD, Lee JH, Pascua CJ, Chen YH, Monteys AM, Davidson BL.  Cardiac mTORC1 dysregulation impacts  stress adaptation and survival in Huntington Disease, Cell Rep 23(4):1020-1033, 2018.  doi:  10.1016/j.celrep.2018.03.117

Amado DA, Rieders JM, Diatta F, Hernandez-Con P, Singer A, Zhang J, Lancaster E, Davidson BL, Chen-Plotkin  AS.  AAV-mediated progranulin delivery to a mouse model of progranulin deficiency causes T cell-mediated  hippocampal degeneration.  bioRxiv 308692. Posted 4/27/18.

Cheng C, Spengler RM, Keiser MS, Monteys AM, Rieders JM, Ramachandran S, Davidson BL.  The long noncoding  RNA NEAT1 is elevated in polyglutamine repeat expansion diseases and protects from disease-gene dependent  toxicities.  Hum Mol Genet 2018, Sept. 19 [Epub ahead of print].

Sun JH, Zhou L, Emerson DJ, Phyo SA, Titus KR, Gong W, Gilgenast TG, Beagan JA, Davidson BL, Tassone F,  Phillips-Cremins JE.  Disease-associated short tandem repeats co-localize with chromatin domain boundaries.  Cell  175(1):224-238, 2018.


Dissen GA, Adachi K, Lomniczi A, Chatkupt T, Davidson BL, Nakai H, Ojeda SR.  Engineering as a gene silencing viral construct that targets the cat hypothalamus to induce permanent sterility: An update.  Reprod Dom Anim 2017 Apr;52 Suppl 2-354-358.  doi: 10.111/rda.12834 [Epub 2016 Nov 17].

Keiser MS, Mas Monteys A, Corbau R, Gonzalez-Alegre P, Davidson BL.  RNAi prevents and reverses phenotypes induced by mutant human ataxin-1.  Annals of Neurology, 2016 Nov; 80(5):754-765.  DOI: 10.1002/ana.24789.

Ramachandran, S, Coffin SL, Tang T-Y, Jobaliya CD, Spengler RM, Davidson BL.  Cis-Acting single nucleotide polymorphisms alter MicroRNA-mediated regulation of human brain expressed transcripts.  Human Mol Genet, 25(22):4939-4950, 2016.

Monteys AM, Ebanks SA, Keiser MS, Davidson BL.  CRISPR/Cas9 editing of the mutant Huntingtin allele in vitro and in vivo.  Mol Ther 25(1):12-23, 2017.

Carmona V, Cunha-Santos J, Onofre I, Simoes AT, Vijayakumar U, Davidson BL, Pereira de Almeida L.  Unravelling endogenous MicroRNAs system dysfunction as a new pathophysiological mechanism in Machado-Joseph disease.  Mol Ther, 25(4):1038-1055, 2017 [Epub 2017 Feb 22].

Kim Y-C, Miller A, Lins LCRF, Han W-W, Keiser MS, Boudreau RL, Davidson BL, Nandakumar S.  RNA interference of human alpha-synuclein in mouse.  Front Neurol 2017 Jan 31. 10.3389/fneur2017.00013.

Machuca TN, Cypel M, Bonato R, Yeung J, Chun Y-M, Juvet S, Guan Z, Hwang DM, Chen M, Saito T, Harmantas C, Davidson BL, Waddell TK, Liu M, Keshavjee S.  Safety and efficacy of ex-vivo donor lung adenoviral IL-10 gene therapy in a large animal lung transplant survival model.  Hum Gene Ther.  January 2017,


Ochaba J, Monteys AM, O'Rourke JG, Reidling JC, Steffan JS, Davidson BL, Thompson LM.  PIAS1 regulates mutant Huntingtin accumulation and Huntington's disease-associated phenotypes in vivo.  Neuron. 2016 May 4;90(30)507-20.

Lin L, Park JW, Ramachandran S, Zhang Y, Tseng YT, Shen S, Waldvogel H, Curtis M, Faull R, Troncoso J, Ross C, Davidson BL*, Xing Y*.  Transcriptome sequencing reveals aberrant alternative splicing in Huntington's disease.  Hum Mol Genet, 2016, 25(16):3454-3466, 2016.  (*joint corresponding authors)

Spengler RM, Zhang X, Cheng C, McLendon JM, Skeie JM, Johnson FL, Davidson BL, Boudreau RL.  Elucidation of transcriptome-wide microRNA binding sites in human cardiac tissues by Ago2HITS-CLIP.  Nucl Acids Research. 2016 Sep 6;44(1):7120-2131, 2016.


Lee JH, Tecedor L, Chen YH, Sowada MJ, Monteys AM, Thompson LM, Davidson BL.  Reinstating aberrant mTORC1 activity in Huntington's disease improves disease phenotypes.   Neuron. 2015 Jan 21;85(2):303-15.  Commentary in: Sci Signal 8, 3 Feb. 2015, 8(362):ec26; Nature Reviews Neuroscience,16:66-67, 2015.

Monteys AM, Wilson MJ, Boudreau RL, Spengler RM and Davidson BL.  Artificial miRNAs targeting mutant huntingtin show preferential silencing in vitro and in vivo. Mol Ther Nucl Acids, 2015; Apr 7;4:e234.

Madhavan L, Daley BF, Davidson BL, Boudreau RL, Cole-Strauss A, Lipton JW, Steece-Collier K, and Collier TJ; Sonic Hedgehog controls the phenotypic fate and therapeutic efficacy of grafted neural precursor cells in a model of nigrostriatal neurodegeneration.  PLOSOne, 2015 Sept 4; 10(9):e0137136.

Keiser MS, Kordower JH, Gonalez-Alegre P, Davidson BL.  Broad distribution of ataxin 1 silencing in rhesus cerebella for spinocerebellar ataxia type 1 therapy.  Brain. 2015 Dec; 138(Pt 12):3555-66.

Katz ML, Tecedor L, Chen Y, William BG, Lysenko E, Wininger FA, Young WM, Johnson GC, Whiting REH, Coates JR, Davidson BL.  AAV gene transfer delays disease onset in a TPP1-deficient canine model of late infantile form of Batten disease.  Sci Transl Med 2015 Nov. 11; 7(313):313ra180.  Highlighted by NINDS, Science Signaling.


Boudreau RL, Jiang P, Gilmore BL, Spengler RM, Tirabassi R, Nelson JA, Ross CA, Xing Y, Davidson BL. Transcriptome-wide discovery of microRNA binding sites in human brain, Neuron, 81(2): 294-305, 2014.

Lee JH, Sowada MJ, Boudreau RL, Aerts AM, Thedens DR, Nopoulos P, Davidson BL. Rhes Suppression Enhances Disease Phenotypes in Huntington's Disease Mice. J Huntingtons Dis. 2014 Jan 1;3(1):65-71.

Spengler RM, Oakley CK, Davidson BL. Functional microRNAs and target sites are created by lineage-specific transposition, Hum Mol Genet, 2014 Apr 1; 23(7):1783-130.

209 Keiser MS, Boudreau RL, Davidson BL. Broad Therapeutic Benefit After RNAi Expression Vector Delivery to Deep Cerebellar Nuclei: Implications for Spinocerebellar Ataxia Type 1 Therapy. Mol Ther 22(3): 588-95, 2014.

McLoughlin HS, Wan J, Spengler RM, Xing Y, Davidson BL. Human-specific microRNA regulation of FOXO1: implications for microRNA recognition element evolution. Hum Mol Genet, 23(10): 2593-2603, 2014.

Griffin MA, Restrepo MS, Abu-El-Haija M, Wallen T, Buchanan E, Rokhlina T, Chen YH, McCray PB Jr, Davidson BL, Divekar A, Uc A. A novel gene delivery method transduces porcine pancreatic duct epithelial cells. Gene Ther. 21(2): 123-30 2014

Schultz ML, Tecedor L, Stein CS, Stamnes MA, Davidson BL.  CLN3 deficient cells display defects in the ARF1-Cdc42 pathway and actin-dependent events.  PLos One 9(5): e96647, 2014.

Ramachandran PS, Bhattarai S, Singh P, Boudreau RL, Thompson S, LaSpada AR, Drack AV, Davidson BL. RNA interference-based therapy for spinocerebellar ataxia type 7 retinal degeneration. PLoS One 9(4): e95362, 2014.

Ramachandran PS, Boudreau RL, Schafer KA, LaSpada AR, Davidson BL. Non-allele specific silencing of ataxin-7 improves disease phenotypes in a mouse model of SCA7.  Mol Ther. 22(9):1635-42, 2014.

Krishnamurthy S, Behlke MA, Apicella MA, McCray PB, Davidson BL.  Platelet activating factor receptor activation improves siRNA uptake and RNAi responses in well-differentiated airway epithelia.  Mol Ther Nucleic Acids. 2014 Jul 15;3:e175. doi: 10.1038/mtna.2014.26.

Puppo A, Cesi G, Marrocco E, Piccolo P, Jacca S, Shayakhmetov DM, Parks RJ, Davidson BL, Colloca S, Brunetti-Pierri N, Ng P, Donofrio G, Auricchio A. Retinal transduction profiles by high-capacity viral vectors. Gene Ther. 21(10):855-865, 2014.

Geoghegan JC, Keiser NW, Okulist A, Martins I, Wilson MS, Davidson BL.  Chondroitin sulfate is the primary receptor for a peptide-modified AAV that targets brain vascular endothelium in vivo.  Mol Ther Nucleic Acids 2014; 3:e202. doi:10.1038/mtna.2014.50.

Monteys A, Spengler A, Dufour B, Wilson M, Oakley C, Sowada M, McBride J, Davidson B.  Single nucleotide seed modification restores in vivo tolerability of a toxic artificial miRNA sequence in the mouse brain.  Nucl Acids Res 42(21):13315-13327, 2014, doi: 10.1093/nar/gku979


Rodriguez-Lebron E, Liu G, Keiser MS, Behlke MA, Davidson BL. Altered Purkinje cell miRNA expression and SCA1 pathogenesis. Neurobiol Dis. 54:456-463. doi: 10.1016/j.nbd.2013.01.019. Epub 2013. 

Ramachandran S, Krishnamurthy S, Jacobi AM, Wohlford-Lenane C, Behlke MA, Davidson BL, McCray PB Jr. Efficient Delivery of RNA-interference Oligonucleotides to Polarized Airway Epithelia In Vitro. Am J Physiol Lung Cell Mol Physiol. 305(1):L23-32, 2013.

Ramachandran PS, Keiser MS, Davidson BL Recent advances in RNA interference therapeutics for CNS diseases.  Neurotherapeutics 10(3):473-485.  doi 10.1007/s13311-013-0183-8, 2013.

Keiser MS, Geoghegan JC, Boudreau RL, Lennox KA, Davidson BL. RNAi or overexpression: Alternative therapies for Spinocerebellar Ataxia Type 1. Neurobiol Dis. 56:6-13, doi:10.1016/j.nbd, 2013.

Cheng C, Chen YH, Lennox KA, Behlke MA, Davidson BL. In vivo SELEX for identification of brain-penetrating aptamers. Mol Ther Nucleic Acids. 2Le67,  doi 10.1038/mtna, 2013.

Benraiss A, Toner MJ, Xu Q, Bruel-Jungerman E, Rogers EH, Wang F, Economides AN, Davidson BL, Kageyama R, Nedergaard M, Goldman SA. Sustained mobilization of endogenous neural progenitors delays disease progression in a transgenic model of Huntington's disease. Cell Stem Cell. 12(6):787-799, 2013.

do Carmo Costa M, Luna-Cancalon K, Fischer S, Ashraf NS, Ouyang M, Dharia RM, Martin-Fishman L, Yang Y, Shakkottai VG, Davidson BL, Rodríguez-Lebrón E, Paulson HL. Toward RNAi Therapy for the Polyglutamine Disease Machado-Joseph Disease. Mol Ther. 21(10) 1898-1908, 2013.

Rodríguez-Lebrón E, Costa MD, Luna-Cancalon K, Peron TM, Fischer S, Boudreau RL, Davidson BL, Paulson HL. Silencing mutant ATXN3 expression resolves molecular phenotypes in SCA3 transgenic mice. Mol Ther.  21(10): 1909-1918, 2013.

O'Rourke JG, Gareau JR, Ochaba J, Song W, Raskó T, Reverter D, Lee J, Monteys AM, Pallos J, Mee L, Vashishtha M, Apostol BL, Nicholson TP, Illes K, Zhu YZ, Dasso M, Bates GP, Difiglia M, Davidson B, Wanker EE, Marsh JL, Lima CD, Steffan JS, Thompson LM. SUMO-2 and PIAS1 modulate insoluble mutant huntingtin protein accumulation. Cell Rep. 4(2):362-75. 2013

Hudry E, Dashkoff J, Roe AD, Takeda S, Koffie RM, Hashimoto T, Scheel M, Spires-Jones T, Arbel-Ornath M, Betensky R, Davidson BL, Hyman BT. Gene transfer of human Apoe isoforms results in differential modulation of amyloid deposition and neurotoxicity in mouse brain. Sci Transl Med.  5(212): 212, 2013.

Tecedor L, Stein CS, Schultz ML, Farwanah H, Sandhoff K, Davidson BL. CLN3 loss disturbs membrane microdomain properties and protein transport in brain endothelial cells. J Neurosci. 33(46):18065-79, 2013.


Chen YH, Claflin K, Geoghegan JC, Davidson BL. Sialic acid deposition impairs the utility of AAV9, but not peptide-modified AAVs for brain gene therapy in a mouse model of lysosomal storage disease. Mol Ther. 20(7):1393-1399, 2012.

Fineberg SK, Datta P, Stein CS, Davidson BL. MiR-34a represses Numbl in murine neural progenitor cells and antagonizes neuronal differentiation. PLoS One. 7(6):e38562, 2012.

McLoughlin HS, Fineberg SK, Ghosh LL, Tecedor L, Davidson BL. Dicer is required for proliferation, viability, migration and differentiation in corticoneurogenesis. Neuroscience. 223-285. Doi10.1016, 2012 

Boudreau RL, Spengler RM, Hylock RH, Kusenda BJ, Davis HA, Eichmann DA, Davidson BL.  siSPOTR: a tool for designing highly specific and potent siRNAs for human and mouse.   Nucleic Acids Research, 4(1)e9 doi: 10.1093/nar/gks797, 2012.

Krishnamurthy S, Behlke MA, Ramachandran S, Salem AK, McCray PB Jr and Davidson BL. Manipulation of Cell Physiology Enables Gene Silencing in Well-differentiated Airway Epithelia.   Mol Ther Nucleic Acids. 1:e41. Doi: 10.1038/mtna, 2012.


Ding SL, Tecedor L, Stein CS, Davidson BL. A knock-in reporter mouse model for Batten disease reveals predominant expression of Cln3 in visual, limbic and subcortical motor structures. Neurobiol Dis. 41(2):237-248, 2011.

Shen S., Lin L., Cai JJ., Jiang P., Kenkel EJ., Stroik MR., Sato S., Davidson BL., Xing Y. Widespread establishment and regulatory impact of Alu exons in human genes.  PNAS. 108(7):2837-2842, 2011.

Boudreau RL, Spengler RM and Davidson BL. Rational Design of Therapeutic siRNAs: Minimizing Off-targeting Potential to Improve the Safety of RNAi Therapy for Huntington’s Disease. Mol Ther.  19(12):2169-2177, 2011.

McBride JL, Pitzer MR, Boudreau RL, Dufour B, Hobbs T, Ojeda SR and Davidson BL. Preclinical safety of RNAi-mediated HTT suppression in the rhesus macaque as a potential therapy for Huntington’s disease. Mol Ther. 19(12): 2152-2162, 2011. 


Monteys AM, Spengler RM, Wan J, Tecedor L, Lennox KA, Xing Y, Davidson BL.  Structure and activity of putative intronic miRNAs promoters.  RNA 16(3): 495-505.  2010.

Ebert SM, Monteys AM, Fox DK, Bongers KS, Shields BE, Malmberg SE, Davidson BL, Suneja M, Adams CM.  The transcription factor ATF4 promotes skeletal myofiber atrophy during fasting.  Mol Endocrinol.  24(4):790-799, 2010. 

Lin L, Shen S, Jiang P, Sato S, Davidson BL, Xing Y. Evolution of alternative splicing in primate brain transcriptomes. Hum Mol Genet. 19(15):2958-2973, 2010.

Da Silva LF, Walder RY, Davidson BL, Wilson SP, Sluka KA.  Changes in expression of NMDA-NR1 receptor subunits in the rostral ventromedial medulla modulate pain behaviors.  Pain. 151(1):155-161. 2010.

Stein CS, Yancey PH, Martins I, Sigmund RD, Stokes JB, Davidson BL. Osmoregulation of ceroid neuronal lipofuscinosis type 3 in the renal medulla. Am J Physiol Cell Physiol. 298(6):C1388-1400. 2010.

Piedrahita D, Hernández I, López-Tobón A, Fedorov D, Obara B, Manjunath BS, Boudreau RL, Davidson BL, Laferla F, Gallego-Gómez JC, Kosik KS, Cardona-Gómez GP. Silencing of CDK5 reduces neurofibrillary tangles in transgenic Alzheimer’s mice.  J Neurosci. 30(42);13966-13976, 2010.


Boudreau RL., McBride JL, Martins I, Shen S, Xing Y, Carter BJ, Davidson BL. Non-allele-specific silencing of mutant and wildtype huntingtin demonstrates therapeutic efficacy in Huntington's disease mice. Mol Ther 17(6):1053-1063, 2009.

Hadaczek P, Forsayeth J, Mirek H, Munson K, Bringas J, Pivirotto P, McBride JL, Davidson BL, Bankiewicz KS.  Transduction of nonhuman primate brain with adeno-associated virus serotype 1: vector trafficking and immune response.  Hum Gene Ther. 20(3):225-237, 2009

Cypel M, Liu M, Rubacha M, Yeung JC, Hirayama S, Anraku M, Sato M, Medin J, Davidson BL, de Perrot M, Waddell TK, Slutsky AS, Keshavjee S. Functional repair of human donor lungs by IL-10 gene therapy. Sci Transl Med. 1(4):4ra9, 2009.

Borchert GM, Gilmore BL, Spengler RM, Xing Y, Lanier W, Bhattacharya D, Davidson BL. Adenosine deamination in human transcripts generates novel microRNA binding sites. Hum Mol Genet. 18(24):4801-4807, 2009.

Lin, L, Jiang, P, Shen, S, Sato, S, Davidson, BL, Xing Y.  Large-scale analysis of exonized mammalian-wide interspersed repeats in primate genomes.  Hum Mol Genet. 18(12):2204-2214, 2009. 

Rodriguez-Lebron E, Gouvion CM, Moore SA, Davidson BL, Paulson HL.  Allele-specific RNAi Mitigates Phenotypic Progression in a Transgenic Model of Alzheimer’s Disease.  Mol Ther. 17(9) 1563-1573. 2009.

Quinn K, Brindley MA, Weller ML, Kaludov N, Kondratowicz A, Hunt CL, Sinn PL, McCray PB Jr, Stein CS, Davidson BL, Flick R, Mandell R, Staplin W, Maury W, Chiorini JA.  Rho GTPases modulate entry of Ebola virus and vesicular stomatitis virus pseudotyped vectors.  J Virol.  83(19):10176-10186, 2009.

Chen, YH, Chang, M, Davidson BL.  Molecular signatures of disease brain endothelia provide new sites for CNS-directed enzyme therapy. Nat Med 15(10):1215-8.  2009.  Comment in Nat Med. 15(10):1215-1218. 2009


Chang M, Cooper JD, Sleat DE, Cheng SH, Dodge JC, Passini MA, Lobel P, Davidson BL.  Intraventricular Enzyme Replacement in a Mouse Model of Late Infantile Neuronal Ceroid Lipofuscinosis.  Mol Ther 16(4):649-56, 2008.

McBride JL, Boudreau RL, Harper, SQ, Staber PD, Monteys AM, Martins I, Gilmore BL, Burstein H, Peluso RW, Polisky B, Carter BJ, Davidson BL.  Artificial miRNAs mitigate shRNA-mediated toxicity in the brain: Implications for the therapeutic development of RNAi.  Proc Natl Acad Sci USA.  105(15):5868-73, 2008.

Boudreau RL, Mas Monteys, A, Davidson, BL.  Minimizing variables among hairpin-based RNAi vectors reveals the potency of shRNAs.  RNA. 14(9):1834-1844, 2008. (Citations: 57)

Lin L, Shihao S, Tye A, Cai JJ, Jiang P, Davidson BL, Xing Y.  Diverse Splicing Patterns of Exonized Alu Elements in Human Tissues.  PloS Genetics. 4(10):e1000225, 2008.

Packer AN, Xing Y, Harper SQ, Jones L, Davidson BL. The bi-functional microRNA miR-9/miR-9* regulates REST and CoREST and is down-regulated in Huntington’s Disease. J Neurosci  28(53):14341-1446., 2008 

Boudreau BL, Martins I, Davidson BL.  Artificial microRNAs as siRNA shuttles: improved safety as compared to shRNAs in vitro and in vivo.  Mol Ther 17(1): 169-175, 2009. (Rated top article of 2009).

Coryell M, Wunsch AM, Haenfler JM, Allen JE, McBride JL, Davidson BL, Wemmie JA.  Restoring Acid-Sensing Ion Channel-1a in the Amygdala of Knockout Mice Rescues Fear Memory but not Unconditioned Fear Responses.  J.  Neurosci. 28(51): 13738-13741, 2008


Liu G, Chen YH, He X, Martins I, Heth JA, Chiorini JA, Davidson BL.  Adeno-associated virus type 5 reduces learning deficits and restores glutamate receptor subunit levels in MPS VII mice CNS.  Mol Ther 15(2):242-247, 2007.

Cabrera-Salazar MA, Roskelley EM, Bu J, Hodges BL, Yew N, Dodge JC, Shihabuddin LS, Sohar I, Sleat DE, Scheule RK, Davidson BL, Cheng SH, Lobel P and Passini MA. Timing of therapeutic intervention determines functional and survival outcomes in a mouse model of late infantile Batten disease. Mol Ther 15(10):1782-8, 2007.

Vibhakar R, Foltz G, Yoon JG, Field L, Lee H, Ryu GY, Pierson J, Davidson BL, Madan A. Dickkoph-1 is an epigenetically silenced candidate tumor suppressor gene in medulloblastoma. Neuro Oncol 9(2):135-44, 2007.

Kumar P, Wu H, McBride JL, Jung KE, Kim MH, Davidson BL, Lee SK, Shankar P, Manjunath N. Transvascular delivery of small interfering RNA to the central nervous system. Nature 448(7149):39-43, 2007. Listed in Faculty of 1000 and highlighted in a commentary by Dr. J. Rossi in Nature.

Eliason SL, Stein CS, Mao Q, Tecedor L, Ding SL, Gaines DM, Davidson BL. A knock-in reporter model of Batten disease. J. Neurosci 27(37):9826-34, 2007.


Passini M, Dodge JC, Bu J, Yang W, Zhao Q, Sondhi D, Hackett NR, Kaminsky SM, Mao Q, Shihabuddin LS, Cheng SH, Sleat DE, Stewart GR, Davidson BL, Lobel P, Crystal RG.  Intracranial delivery of CLN2 reduces brain pathology in a mouse model of classical late infantile neuronal ceroid lipofuscinosis. J Neurosci 26(5):1334-1342, 2006.

Harper SQ, Staber PD, Beck CR, Fineberg SK, Stein CS, Ochoa D, Davidson BL.  Optimization of FIV vectors for RNA interference.  J Virol 80(19):9371-9380, 2006.

Kang Y, Moressi CJ, Scheetz TE, Xie L, Tran DT, Casavant TL, Ak P, Benham CJ, Davidson BL, McCray PB Jr.  Integration site choice of a feline immunodeficiency virus vector.  J Virol 80(19):8820-8823, 2006

Borchert GM, Lanier W, Davidson BL.  RNA polymerase III transcribes human microRNAs.  Nat Struct Mol Biol 13(12):1097-1101, 2006 – The article of the month in this journal, and listed in Faculty of 1000.

Allaire PD, Ritter B, Thomas S, Burman JL, Denisov AY, Legendre-Guillemin V, Harper SQ, Davidson BL, Gehring K, McPherson PS.  Connecdenn, a novel DENN domain-containing protein of neuronal clathrin-coated vesicles functioning in synaptic vesicle endocytosis.  J. Neurosci. 26(51):13202-12, 2006 


Stein CS, Martins I, Davidson BL.  The lymphocytic choriomeningitis virus envelope glycoprotein targets lentiviral gene transfer vector to neural progenitors in the murine brain.  Mol Ther 11(3):382-389, 2005.

Harper SQ, Staber PD, He X, Eliason SL, Martins IH, Mao Q, Yang L, Kotin RM, Paulson BL, Davidson BL.  RNA interference improves motor and neuropathological abnormalities in a Huntington’s disease mouse model.  PNAS 102(16):5820-5825, 2005. Listed in Faculty of 1000. 

Orii KO, Grubb JH, Vogler C, Levy B, Tan Y, Markova K, Davidson BL, Mao Q, Orii T, Kondo N, Sly WS.  Defining the pathway for Tat-mediated delivery of beta-glucuronidase in cultured cells and MPS VII mice.  Mol Ther 12(2):345-352, 2005.

Kang Y, Xie L, Tran DT, Stein CS, Hickey M, Davidson BL, McCray PB Jr.  Persistent expression of factor VIII in vivo following nonprimate lentiviral gene transfer.  Blood 106(5):1552-1558, 2005.

Liu G, Martins IH, Chiorini JA, Davidson BL.  Adeno-associated virus type 4 (AAV4) targets ependyma and astrocytes in the subventricular zone and RMS.  Gene Therapy 12(20):1503-1508, 2005.

Liu G, Martins I, Wemmie JA, Chiorini JA, Davidson BL. Functional Correction of CNS Phenotypes in A Lysosomal Storage Disease Model using Adeno-associated Virus Type 4 Vectors. J Neurosci 25(41):9321-9327, 2005. A highlighted article in J. Neuroscience. 


Miller VM, Gouvion CM, Davidson BL, Paulson HL.  Targeting Alzheimer’s disease genes with RNA interference: an efficient strategy for silencing mutant alleles.  Nucleic Acids Res 32(2):661-668, 2004.

Sinnayah P, Lindley TE, Staber PD, Davidson BL, Cassell MD, Davisson RL.  Targeted viral delivery of Cre recombinase induces conditional gene deletion in cardiovascular circuits of the mouse brain.  Physiol Genomics 18(1):25-32, 2004.

Xia H, Mao Q, Eliason SL, Harper SQ, Martins IH, Orr HT, Paulson HL, Yang L, Kotin RM, Davidson BL.  RNAi suppresses polyglutamine-induced neurodegeneration in a model of spinocerebellar ataxia.  Nat Med 10(8):816-820, 2004. Listed in Faculty of 1000. 

Sleat EA, Wiseman JA, El-Banna M, Kim KH, Mao Q, Price S, Macauley SL, Sidman RL, Shen MM, Zhao Q, Passini MA, Davidson BL, Stewart GR, Lobel P.  A mouse model of classical late-infantile neuronal ceroid lipofuscinosis based on targeted disruption of the CLN2 gene results in a loss of tripeptidyl-peptidase I activity and progressive neurodegeneration.  J Neurosci 24(41):9117-9126, 2004.

Martins S, de Perrot M, Imai Y, Yamane M, Quardri SM, Segall L, Dutly A, Sakiyama S, Chaparro A, Davidson BL, Waddell TJ, Liu M, Keshavjee S.  Transbronchial administration of adenoviral-mediated interleukin-10 gene to the donor improves function in a pig lung transplant model.  Gene Ther 11(24):1786-1796, 2004.


Haskell RE, Hughes SM, Chiorini JA, Alisky JM, Davidson BL.  Viral-mediated delivery of the late-infantile neuronal ceroid lipofuscinosis gene, TPP I to the mouse central nervous system.  Gene Ther 10(1):34-42, 2003.

de Perrot M, Fischer S, Liu M, Imai Y, Martins S, Sakiyama S, Tabata T, Bai XH, Waddell TK, Davidson BL, Keshavjee S.  Impact of human interleukin-10 on vector induced inflammation and early graft function in rat lung transplantation.  Am J Resp Cell & Mol Biol 28(5):616-625, 2003.

Mao Q, Foster BJ, Xia, H, Davidson BL.  Membrane topology of CLN3, the protein underlying Batten Disease.  FEBS Lett 541(1-3):40-46, 2003.

Kim M, Mao Q, Davidson BL, Wiemer, DF.  Tripeptide probes for tripeptidyl protease I production via gene transfer.  J Med Chem 46(9):1603-1608, 2003.

Sinn PL, Hickey MA, Staber PD, Dylla DE, Jeffers DA, Davidson BL, Sanders DA, McCray PB Jr.  Lentivirus vectors pseudotyped with filoviral envelope glycoproteins transduce  airway epithelia from the apical surface independently of folate receptor alpha.  J Vriol 77(10):5902-5910, 2003

Miller VM, Xia H, Marrs GL, Gouvion CM, Lee G, Davidson BL, Paulson HL.  Allele-specific silencing of dominant disease genes.  Proc Natl Acad Sci, USA 100(12):7195-7200, 2003.

Gonzalez-Alegre P, Miller VM, Davidson BL, Paulson HL.  Toward therapy for DYT1 dystonia: allele-specific silencing of mutant TorsinA.  Ann Neurol 53(6):781-787, 2003.

Davidson BL, XO Breakefield.  Viral vectors for gene delivery to the nervous system.  Nat Rev Neurosci 4(5):353-364, 2003.

Elzey BD, Tian J, Jensen RJ, Swanson AK, Lees JR, Lentz SR, Stein CS, Nieswandt B, Wang Y, Davidson BL, Ratliff TL.  Platelet-mediated modulation of adaptive immunity.  A communication link between innate and adaptive immune compartments.  Immunity 19(1):9-19, 2003.

Alisky JM, Hughes SM, Davidson BL.  Transduction of neurons lining the cerebral external capsules in mice with feline immunodeficiency virus based vectors.  Neurosci Lett 351(2):120-124, 2003.

Mao Q, Xia H, Davidson BL.  Intracellular trafficking of CLN3, the protein underlying the childhood neurodegenerative disease, Batten disease.  FEBS Lett 555(2):351-357, 2003.

DiPasquale G, Davidson BL, Stein CS, Martins I, Scudiero D, Monks A, Chiorini JA.  Identification of PDGFR as a receptor for AAV-5 transduction.  Nat Med 9(10):1306-1312, 2003.

Lotery AJ, Yang GS, Mullins RF, Russell SR, Schmidt M, Stone EM, Lindbloom JD, Chiorini JA, Kotin RM, Davidson BL.  Adeno-associated virus type 5: transduction efficiency and cell-type specificity in the primate retina.  Hum Gene Ther 14(17):1663-1671, 2003.


Rauma T, Kumpumäki S, Anderson R, Davidson BL, Ruotsalainen H, Myllylä R, Hautala T.  Adenoviral gene transfer restores lysyl hydroxylase activity in type VII Ehlers-Danlos syndrome.  J Invest Dermatol 116(4):602-605, 2001

Law L, Davidson BL.  Adenovirus serotype 30 fiber does not mediate transduction via the coxsackie adenovirus receptor.  J Virol 76(2)656-661, 2002.

Hughes SM, Moussavi-Harami F, Sauter SL, Davidson BL.  Viral-mediated gene transfer to mouse primary neural progenitor cells.  Mol Ther 5(1):16-24, 2002

Sinnayah P, Lindley TE, Staber PD, Cassell MD, Davidson BL, Davisson RL.  Selective gene transfer to key cardiovascular regions of the brain: comparison of two viral vector systems.  Hypertension 39(2 Pt 2):603-608, 2002

Lotery AJ, Derksen TA, Russell SR, Mullins RF, Sauter SL, Affatigato LM, Stone EM, Davidson BL.  Gene transfer to the non-human primate retina with recombinant feline immunodeficiency virus vectors.  Hum Gene Ther 13(6):689-696, 2002.

Abounader R, Lal B, Luddy C, Koe G, Davidson BL, Rosen EM, Laterra J.  In vivo targeting of SF/HGF and C-MET expression via U1snRNA/ribozymes inhibits glioma growth and angiogenesis and promotes apoptosis.  FASEB J 16(1):108-110, 2002.

Seigel GM, Lotery A, Kummer A, Bernard D, Green ND, Turmaine M, Derksen T, Nussbaum RL, Davidson BL, Wagner J, Mitchison HM.  Retinal pathology and function in a CLN3-knockout mouse model of juvenile Neuronal  Ceriod Lipofuscinosis (Batten Disease).  Mol Cell Neurosci Mol & Cell Neurosci 19(4):515-527, 2002

Derksen TA, Sauter SL, Davidson BL.  Feline immunodeficiency virus vectors.  Gene transfer to mouse retina following intravitreal injection.  J Gene Med 4(5)463-469, 2002. (Citations: 20)

Wang G, Williams G, Xia H, Hickey M, Shao J, Davidson BL, McCray PB Jr.  Apical barriers to airway epithelial cell gene transfer with amphotropic retroviral vectors.  Gene Ther 9(14):922-931, 2002.

Brooks AI, Stein CS, Hughes SM, Heth J, McCray PB Jr, Sauter SL, Johnston JC, Cory-Slechta DA, Federoff HJ, Davidson BL.  Functional correction of established CNS deficits in an animal model of lysosomal storage disease using feline immunodeficiency virus-based vectors.  Proc Natl Acad Sci, USA 99(9):6216-6221, 2002. Highlighted in a commentary by Drs. Sly and Vogler in the same issue. (Citations: 109)

Kang Y, Stein CS, Heth JA, Sinn PL, Penisten AK, Staber PD, Shen H, Barker CK, Martins I, Sharkey CM, Sanders DA, McCray PB Jr, Davidson BL.  In vivo gene transfer using a nonprimate lentiviral vector pseudotyped with Ross River virus glycoproteins.  J Virol 76(18):9378-9388, 2002.

Yang GS, Schmidt M, Yan Z, Lindbloom JD, Harding TC, Donahue BA, Engelhardt JF, Kotin RM, Davidson BL.  Viral mediated transduction of murine retina with AAV: effects of viral capsid and genome size.  J Virol 76(15):7651-7660, 2002.

Xia H, Mao Q, Paulson HL, Davidson BL.  SiRNA-mediated gene silencing with recombinant viral vectors.  Nat Biotech 20:1006-1010, 2002.Commentaries accompanied this paper. This was listed in Faculty of 1000.

Alisky JM, van de Wetering CI, Davidson BL.  Widespread dispersal of cholera toxin subunit b to brain and spinal cord neurons following systemic delivery.  Exp Neurol 178(1)139-146, 2002.


Chu Y, Heistad DD, Cybulsky MI, Davidson BL.  VCAM-1 augments adenovirus-mediated gene transfer.  Arterioscler Thromb Vasc Biol 21(2):238-242, 2001.

Vasquez EC, Beltz TG, Haskell RE, Johnson RF, Meyrelles SS, Davidson BL, Johnson AK.  Adenovirus-mediated gene delivery to cells of the magnocellular hypothalamo-neurohypophyseal system.  Exp Neurol 167(2):260-271, 2001.

Slepushkin VA, Staber PD, Wang G, McCray PB Jr. Davidson BL.  Infection of human airway epithelia with H1N1, H2N2, and H3N2 influenza A virus strains.  Mol Ther 3(3):395-402, 2001.

Jacobson N, Andrews M, Shepard AR, Nishimura D, Searby C, Fingert JH, Hageman G, Mullins R, Davidson BL, Kwon YH, Alward WL, Stone EM, Clark AF, Sheffield VC.  Non-secretion of mutant proteins of the glaucoma gene myocilin in cultured trabecular meshwork cells and in aqueous humor.  Hum Mol Genet 10(2):117-125, 2001.

Stein CS, Kang Y, Sauter SL, Townsend K, Staber P, Derksen TA, Martins I, Qian J, Davidson BL, McCray PB Jr.  In vivo treatment of hemophilia A and mucopolysaccharidosis type VII using non-primate lentiviral vectors.  Mol Ther 3(6):850-856, 2001.

Xia H, Mao Q, Davidson BL. The HIV Tat protein transduction domain improves the biodistribution of  beta-glucuronidase expressed from recombinant viral vectors. Nat Biotech 19(7):640-644, 2001.


Stein CS, Martins I, Davidson BL.  Long term reversal of hypercholesterolemia in low density lipoprotein receptor (LDLR)-deficient mice by adenovirus-mediated LDLR gene transfer combined with CD154 blockade.  J Gene Med 2(1):41-51, 2000.

Wang G, Zabner J, Slepushkin V, Deering C, Shao J, Bodner M, Jolly DJ, Davidson BL, McCray PB Jr.  Increasing epithelial junction permeability enhances gene transfer to airway epithelia in vivo.  Am J Respir Cell & Mol Biol 22(2):129-138, 2000.

Durbeej M, Cohn RD, Hrstka RF, Moore SA, Allamand V, Davidson BL, Williamson RA, Campbell KP.  Disruption of the ß-sarcoglycan gene reveals pathogenetic complexity of limb-girdle muscular dystrophy type 2E.  Mol Cell 5(1):141-151, 2000.

Toyoda K, Faraci FM, Russo AF, Davidson BL, Heistad DD.  Gene transfer of calcitonin gene-related peptide to cerebral arteries.  Am J Physiol Heart Circ Physiol 278(2):H586-594,  2000.

Haskell RE, Carr CJ, Pearce DA, Bennett MJ, Davidson BL.  Batten Disease:  Evaluation of CLN3 mutations on protein localization and function.  Hum Mol Gen 9(5):735-744, 2000.

Davidson BL, Stein CS, Heth JA, Martins I, Kotin RM, Derksen, TA, Zabner J, Ghodsi A, Chiorini JA.  Recombinant adeno-associated virus type 2, 4 and 5 vectors:  transduction of variant cell types and regions in the mammalian CNS.  Proc Natl Acad Sci, USA 97(7):3428-3432, 2000.

Zabner J, Seiler M, Walters R, Kotin RM, Fulgerus W, Davidson BL, Chiorini JA.  Adeno-associated virus type 5 (AAV5) but not AAV2 binds to the apical surfaces of airway epithelia and facilitates gene transfer.  J Virol 74(8):3852-3858, 2000.

Leone P, McPhee SWJ, Janson CG, Davidson BL, Freese A, During MJ.  Multi-site partitioned delivery of human tyrosine hydroxylase gene with phenotypic recovery in Parkinsonian rats.  NeuroReport 11(6):1145-1151, 2000

Anderson RD, Haskell RE, Xia H, Roessler BJ, Davidson BL. A simple method for the rapid generation of recombinant adenovirus vectors.  Gene Ther 7(12):1034-1038, 2000.

Lam EW, Hammond HM, Zwacka R, Darby CJ, Baumgardner KR, Davidson BL, Oberley TD, Engelhardt JF, Oberley LW.  Immunolocalization and adenoviral vector-mediated manganese superoxide dismutase gene transfer to experimental oral tumors.  J Dent Res 79(6):1410-1417, 2000.

Allamand V, Donahue KM, Straub V, Davisson RL, Davidson BL, Campbell KP.  Early adenovirus-mediated gene transfer effectively prevents muscular dystrophy in alpha-sarcoglycan-deficient mice.  Gene Ther 7(16):1385-1391, 2000.

Alisky JM, Hughes SM, Sauter SL, Jolly DJ, Dubensky TW Jr, Staber PD, Chiorini JA, Davidson BL.  Transduction of murine cerebellar neurons with recombinant FIV and AAV5 vectors.  NeuroReport 11(12):2669-2673, 2000.

Griffith TS, Anderson RD, Davidson BL, Williams RD, Ratliff TL.  Adenoviral-mediated transfer of the TNF-related apoptosis-inducing ligand/Apo-2 ligand gene induces tumor cell apoptosis.  J Immunol 165(5):2886-2894, 2000

Xia H, Anderson B, Mao Q, Davidson BL.  Recombinant human adenovirus: targeting to the human transferrin receptor improves gene transfer to brain microcapillary endothelium.  J Virol 74(23):11359-11366, 2000.

Wang G, Deering C, Macke M, Shao J, Burns R, Blau DM, Holmes KV, Davidson BL, Perlman S, McCray PB Jr.  Human coronavirus 229E infects polarized airway epithelia from the apical surface.  J Virol 74(19):9234-9239, 2000.


Chillon M, Bosch A, Zabner J, Law L, Armentano D, Welsh MJ, Davidson BL.  Group D adenoviruses infect primary central nervous system cells more efficiently than those from group C.  J Virol 73(3):2537-2540, 1999.

Stein CS, Derksen TA, Ghodsi A, Davidson BL.  Systemic and central nervous system correction of lysosomal storage in mucopolysaccharidosis type VII mice.  J Virol 73(4):3424-3429, 1999.

Wang G, Slepushkin VA, Bodner M, Zabner J, van Es HH, Thomas P, Jolly DJ, Davidson BL, McCray PB Jr.  Keratinocyte growth factor induced epithelial proliferation facilitates retroviral-mediated gene transfer to pulmonary epithelia in vivo.  J Gene Med 1(1):22-30, 1999.

Lund DD, Faraci FM, Ooboshi H, Davidson BL, Heistad DD.  Adenovirus-mediated gene transfer is augmented in basilar and carotid arteries of heritable hyperlipidemic rabbits.  Stroke 30(1):120-125, 1999.

Haskell RE, Derksen TA, Davidson BL.  Intracellular trafficking of the JNCL protein CLN3.  Mol Genet Metab 66(4):253-260, 1999.

Bohn MC, Choi-Lundberg DL, Davidson BL, Leranth C, Kozlowski DA, Smith JC, O’Banion MK, Redmond DE Jr.  Adenovirus-mediated transgene expression in non-human primate brain.  Hum Gene Ther 10(7):1175-1184, 1999.

Johnston JC, Gasmi M, Lim LE, Elder JH, Yee J-K, Jolly DJ, Campbell KP, Davidson BL, Sauter SL.  Minimal requirements for efficient transduction of dividing and nondividing cells by feline immunodeficiency virus vectors.  J Virol 73(6):4991-5000, 1999.

Stec DE, Davisson RL, Haskell RE, Davidson BL, Sigmund CD.  Efficient liver-specific deletion of a floxed human angiotensinogen transgene by adenoviral delivery of CRE-recombinase in vivo.  J Biol Chem 274(30):21285-21290, 1999.

Lawrence MS, Foellmer HG, Elsworth JD, Kim JH, Leranth C, Kozlowski DA, Bothwell AL, Davidson BL, Bohn MC, Redmond DE Jr.  Inflammatory responses and their impact on ß-galactosidase transgene expression following adenovirus vector delivery to the primate caudate nucleus.  Gene Ther 6(8):1368-1379, 1999.

Ghodsi A, Stein C, Derksen T, Martins I, Anderson RD, Davidson BL.  Systemic hyperosmolality improves -glucuronidase distribution and pathology in murine MPS VII brain following intraventricular gene transfer.  Exp Neurol 160(1):109-116, 1999.

Matthews CB, Jenkins G, Hilfinger JM, Davidson BL.  Poly-L-lysine improves gene transfer with adenovirus formulated in PLGA microspheres.  Gene Ther 6(9):1558-1564, 1999.

Christenson SD, Lund D, Ooboshi H, Faraci FM, Davidson BL, Heistad DD.  Approaches to enhance expression after adenovirus-mediated gene transfer to the carotid artery.  Endothelium 7(1):75-82, 1999.

Connor B, Kozlowski DA, Schallert T, Tillerson JL, Davidson BL, Bohn MC.  The differential effects of adenoviral vector mediated glial cell line-derived neurotrophic factor (GDNF) in the striatum vs substantia nigra of the aged Parkinsonian rat.  Gene Ther 6(12):1936-1951, 1999.

Vasquez EC, Beltz TG, Haskell RE, Johnson RF, Meyrelles SS, Davidson BL, Johnson AK.  Adenovirus-mediated gene delivery to Hypothalamic Magnocellular Neurons in Mice.  Hypertension 34(4):756-761, 1999.

Lam EW, Zwacka R, Seftor EA, Nieva DR, Davidson BL, Engelhardt JF, Hendrix MJ, Oberley LW.  Effects of antioxidant enzyme overexpression on the invasive phenotype of hamster cheek pouch carcinoma cells.  Free Radic Biol Med 27(5-6):572-579, 1999.

Wang G, Slepushkin V, Zabner J, Keshavjee S, Johnston JC, Sauter SL, Jolly DJ, Dubensky T, Davidson BL, McCray PB Jr.  Feline immunodeficiency virus vectors persistently transduce non-dividing airway epithelia in vivo and correct the CF defect. J Clin Invest 104(11):R55-R62, 1999.


Betz AL, Shakui P, Davidson BL.  Gene transfer to rodent brain with recombinant adenoviral vectors: effects of infusion parameters, infectious titer and virus concentration on transduction volume. Exp Neurol 150(1):136-142, 1998.

Brooks AI, Halterman MW, Chadwick CA, Davidson BL, Haak-Frendscho M, Radel C, Porter C, Federoff HJ.  Reproducible and efficient murine CNS gene delivery using a microprocessor-controlled injector.  J Neurosci Methods 80(2):137-147, 1998.

Stein CS, Pemberton J, van Rooijen N, Davidson BL.  Effects of macrophage depletion and anti-CD40 ligand on transgene expression and redosing with recombinant adenovirus.  Gene Ther 5(4):431-439, 1998.

Holt KH, Lim LE, Straub V, Venzke DP, Duclos F, Anderson RD, Davidson BL, Campbell KP.  Functional rescue of the sarcoglycan complex in the BIO 14.6 hamster using delta-sarcoglycan gene transfer.  Mol Cell 1(16):841-848, 1998.

Miller FJ, Gutterman DD, Rios CD, Heistad DD, Davidson BL.  Superoxide production in vascular smooth muscle contributes to oxidative stress and impaired relaxation in atherosclerosis. Circ Res 82(12):1298-1305, 1998.

Christenson SD, Lake KD, Ooboshi H, Faraci FM, Davidson BL, Heistad DD.  Adenovirus mediated gene transfer in vivo to CBV’s and perivascular tissue in mice.  Stroke 29(7):1411-1416, 1998

Beer SJ, Matthews CB, Stein CS, Ross BD, Hilfinger JM, Davidson BL.  Poly(lactic-glycolic) acid copolymer encapsulation of recombinant adenovirus reduces immunogenicity in vivo.  Gene Ther 5(6):740-746, 1998.

Bosch A, McCray PB, Walters KS, Bodner M, Jolly DJ, van Es HH, Nakamura T, Matsumoto K, Davidson BL.  Effects of keratinocyte and hepatocyte growth factor in vivo:  implications for retroviral-mediated gene transfer to liver.  Hum Gene Ther 9(12):1747-1754, 1998.

Rubsam LZ, Davidson BL, Shewach DS.  Superior cytotoxicity with ganciclovir compared with acyclovir and 1-beta-D-arabinofuranosylthymine in herpes simplex virus thymidine kinase-expressing cells: A novel paradigm for cell killing. Cancer Res 58(17):3873-3882, 1998. 

Duclos F, Straub V, Moore SA, Venzke DP, Hrstka RF, Crosbie RH, Durbeej M, Lebakken CS, Ettinger AJ, van der Meulen J, Holt KH, Lim LE, Sanes JR, Davidson BL, Faulkner JA, Williamson R, Campbell KP.   Progressive muscular dystrophy in alpha-sarcoglycan deficient mice. J Cell Biol 142(6):1-11, 1998.

Wang G, Williamson R, Mueller G, Thomas P, Davidson BL, McCray PB Jr.  Ultra-sound-guided gene transfer to hepatocytes in utero.  Fetal Diagn Ther 13(4):197-205, 1998.

Wang G, Davidson BL, Melchert P, Slepushkin VA, van Es HH, Bodner M, Jolly DJ, McCray PB Jr.  Influence of cell polarity on retroviral-mediated gene transfer to differentiated human airway epithelia.  J Virol 72(12):9812-9826, 1998.

Ghodsi A, Yang G, Derksen T, Stein C, Anderson RD, Davidson BL.  Extensive beta-glucuronidase activity in murine CNS after adenovirus-mediated gene transfer to brain. Hum Gene Ther 9(16):2331-2340, 1998.

Wilson ME, Young BM, Davidson BL, Mente KA, McGowan SE.  The importance of TGF-beta in murine visceral leishmaniasis.  J Immunol 161(11):6148-6155, 1998.

Gudmundsson G, Bosch A, Davidson BL, Berg DJ, Hunninghake GW.  Interleukin-10 modulates the severity of hypersensitivity pneumonitis.  Am J Respir Cell Mol Biol 1(5):812-818, 1998.

Toyoda K, Ooboshi H, Chu Y, Fasbender A, Davidson BL, Welsh MJ, Heistad DD.  Cationic polymer and lipids enhance adenovirus-mediated gene transfer to carotid artery.  Stroke 29(10):2181-2188, 1998. (Citations: 36)

Choi-Lundberg DL, Lin Q, Schallert T, Crippens D, Davidson BL, Chang YN, Chiang YL, Qian J, Bardwaj L, Bohn MC.  Behavioral and cellular protection of rat dopaminergic neurons by an adenoviral vector encoding glial cell line-derived neurotrophic factor (GDNF).  Exp Neurol 154(2):261-275, 1998.

Vasquez EC, Johnson RF, Beltz RG, Haskell RE, Davidson BL, Johnson AK.  Replication-deficient adenovirus vector transfer to gfp reporter gene into supraoptic nucleus and subfornical organ neurons.  Exp Neurol 154(2):353-365, 1998.


Choi-Lundberg DL, Lin Q, Chang YN, Chiang YL, Hay CM, Mohajeri H, Davidson BL, Bohn MC.  Dopaminergic neurons protected from degeneration by GDNF gene therapy.  Science 275(5301):838-841, 1997.

Lamping KG, Rios CD, Chun JA, Ooboshi H, Davidson BL, Heistad DD.  Intrapericardial administration of adenovirus for gene transfer.  Am J Physiol  272(1 Pt2):H310-H317, 1997

Fasbender A, Zabner J, Chillón M, Moninger TO, Puga AP, Davidson BL, Welsh MJ.  Complexes of adenovirus with polycationic polymers and cationic lipids increase the efficiency of gene transfer in vitro and in vivo.  J Biol Chem 272(10):6479-6489, 1997.

Davidson BL, Bohn MC.  Recombinant adenovirus. A gene transfer vector for study and treatment of CNS diseases.  Exp Neurol 144(1):125-130, 1997

Muhonen MG, Ooboshi H, Welsh MJ, Davidson BL, Heistad DD.  Gene transfer to cerebral blood vessels after subarachnoid hemorrhage.  Stroke 28(4):822-829, 1997.

McCray PB, Wang G, O’Brien L, Davidson BL, Thomas P.  Proliferation indices of pulmonary epithelia during human and ovine lung development: Gene transfer targets for integrating vectors.  Cell Vision 4(1):42-49, 1997.

Walter E, Croyle MA, Davidson BL, Roessler BJ, Hilfinger JM, Amidon GL.  Adenovirus mediated gene transfer to intestinal epithelial cells as a potential approach for oral delivery of peptides and proteins.  J Controlled Release 46(1-2):75-87, 1997.

Ooboshi H, Chu Y, Rios CD, Faraci FM, Davidson BL, Heistad DD.  Altered vascular function after adenovirus-mediated overexpression of endothelial nitric oxide synthase.  Am J Physiol 273(1 Pt2):H265-H270, 1997.

Yang GY, Zhao YJ, Davidson BL, Betz AL.  Overexpression of interleukin-1 receptor antagonist in the mouse brain reduces ischemic brain injury.  Brain Res 751(2):181-188, 1997.

McCray PB, Wang G, Kline JN, Zabner J, Chada S, Jolly DJ, Chang SM, Davidson BL.  Alveolar macrophages inhibit retroviral-mediated gene transfer to airway epithelia.  Hum Gene Ther 8(9):1087-1093, 1997.

Ooboshi H, Rios CD, Chu Y, Christenson SD, Faraci FM, Davidson BL, Heistad DD.  Augmented adenovirus-mediated gene transfer to atherosclerotic vessels.  Arterioscler Throb Vasc Biol. 17(9):1786-1792, 1997.

Meyrelles SS, Sharma RV, Whiteis CA, Davidson BL, Chapleau MW.  Adenovirus-mediated gene transfer to cultured nodose sensory neurons. Brain Res  Mol Brain Res 51(1-2):33-41, 1997.

Lam EW, Zwacka R, Engelhardt JF, Davidson BL, Domann FE Jr, Yan T, Oberley LW.  Adenovirus-mediated manganese superoxide dismutase gene transfer to hamster cheek pouch carcinoma cells.  Cancer Res 57(24):5550-5556, 1997. (Citations: 40)

O’Connor WM, Davidson BL, Kaplitt MG, Abbey MV, During MJ, Leone P, Langer D, O’Connor MJ, Freese A.  Adenovirus vector-mediated gene transfer into human epileptogenic brain slices:  prospects for gene therapy in epilepsy.  Exp Neurol 148(1):167-178, 1997


Plumb TJ, Bosch A, Roessler BJ, Shewach DS, Davidson BL.  Hypoxanthine-guanine phosphoribosyltransferase (HPRT) expression in the central nervous system of HPRT deficient mice following adenoviral mediated gene transfer.  Neurosci Lett 214(2-3):159-162, 1996.

Goebel EA, Davidson BL, Zabner J, Graham SM, Kern JA.  Adenovirus-mediated gene therapy for head and neck squamous cell carcinomas.  Ann Otol Rhinol Laryngol 105(7):562-567, 1996.

Freese A, During MJ, Davidson BL, Gennarelli TA, Kaplitt MG, Flamm ES, Snyder PJ.  Transfection of human lactotroph adenoma cells with an adenovirus vector expressing tyrosine hydroxylase decreases prolactin release.  J Clin Endocrinol Metab 81(6):2401-2404, 1996.

Fang J, Zhu YY, Smiley E, Bonadio J, Rouleau JP, Goldstein SA, McCauley LK, Davidson BL, Roessler BJ.  Stimulation of new bone formation by direct transfer of osteogenic plasmid genes.  Proc Natl Acad Sci U S A 93(12):5753-5758, 1996.

Hagan P, Barks JD, Yabut M, Davidson BL, Roessler BJ, Silverstein FS.  Adenovirus-mediated over-expression of interleukin-1 receptor antagonist reduces susceptibility to excitotoxic brain injury in perinatal rats.  Neuroscience 75(4):1033-1045, 1996.

Yao SN, Farjo A, Roessler BJ, Davidson BL, Kurachi K.  Adenovirus-mediated transfer of human factor IX gene in immunodeficient and normal mice: Evidence for prolonged stability and activity of the transgene in liver.  Viral Immunol 9(3):141-153, 1996.

Plumb TJ, Bosch A, Roessler BJ, Shewach DS, Davidson BL.  Hypoxanthine-guanine phosphoribosyltransferase (HPRT) expression in the central nervous system of HPRT deficient mice following adenoviral mediated gene transfer.  Neurosci Lett 214(2-3):159-162, 1996.

Welling TH, Davidson BL, Zelenock JA, Stanley JC, Gordon D, Roessler BJ, Messina LM.  Systemic delivery of interleukin-1 receptor antagonist protein using a new strategy of direct adenoviral-mediated gene transfer to skeletal muscle capillary endothelium in the isolated rat hindlimb.  Hum Gene Ther 7(15):1795-1802, 1996.

Bosch A, McCray PB Jr, Chang SM, Ulich TR, Simonet WS, Jolly DJ, Davidson BL.  Proliferation induced by keratinocyte growth factor enhances in vivo retroviral-mediated gene transfer to mouse hepatocytes.  J Clin Invest 98(12):2683-2687, 1996.


Doran SE, Ren XD, Betz AL, Pagel MA, Neuwelt EA, Roessler BJ, Davidson BL.  Gene expression from recombinant viral vectors in the Central Nervous System after blood-brain barrier disruption.  Neurosurgery 36: (5)965-970, 1995.

Roessler BJ, Hartman JW, Vallance DK, Latta JM, Janich S, Davidson BL.  Inhibition of IL-1 induced effects in synoviocytes transduced with the human IL-1 receptor antagonist cDNA using an adenoviral vector.  Hum Gene Ther  6(3):307-316, 1995.

Smythe WR, Hwang HC, Elshami AA, Amin KM, Eck SL, Davidson BL, Wilson JM, Kaiser LR, Albelda SM.  Treatment of experimental human mesothelioma using adenovirus transfer of the herpes simplex thymidine kinase gene.  Ann Surg 222(1):78-86, 1995.

Betz AL, Yang GY and Davidson BL.  Attenuation of stroke size in rats using an adenoviral vector to induce overexpression of interleukin-1 receptor antagonist in brain.  J Cereb Blood Flow Metab 15(4):547-551, 1995.

McCoy RD, Davidson BL, Roessler BJ, Huffnagle GB, Simon RH.  Expression of human interleukin-1 receptor antagonist in mouse lungs using a recombinant adenovirus:  Effects on vector-induced inflammation.  Gene Ther 2(7):437-442, 1995.

Ooboshi H, Welsh MJ, Rios CD, Davidson BL, Heistad DD.  Adenovirus-mediated gene transfer in vivo to cerebral blood vessels and perivascular tissue.  Circ Res 77(1):7-13, 1995.

Li T and Davidson BL.  Phenotype correction of retinal pigment epithelium in murine mucopolysaccharidosis VII by adenovirus-mediated gene transfer.  Proc Natl Acad Sci U S A 92(17):7700-7704, 1995.

Ross BD, Kim B, Davidson BL.  Assessment of ganciclovir toxicity to experimental intracranial gliomas following recombinant adenoviral-mediated transfer of the herpes simplex virus thymidine kinase gene by magnetic resonance imaging and proton magnetic resonance spectroscopy.  Clin Cancer Res 1(6):651-657, 1995.

Chen SJ, Wilson JM, Vallance DK, Hartman JW, Davidson BL, Roessler BJ.  A recombinant adenoviral vector expressing a soluble form of VCAM-1 inhibits VCAM-1/VLA-4 adhesion in transduced synoviocytes.  Gene Ther 2(7):469-480, 1995.

Nilaver G, Muldoon LL, Kroll RA, Pagel MA, Breakefield XO, Davidson BL, Neuwelt EA.  Delivery of herpesvirus and adenovirus to nude rat intracerebral tumors following osmotic blood-brain barrier disruption.  Proc Natl Acad Sci U S A 92(21):9829-9833, 1995.

McCoy RD, Davidson BL, Roessler BJ, Huffnagle GB, Janich SL, Laing TJ, Simon RH.  Pulmonary inflammation induced by incomplete or inactivated adenoviral particles.  Hum Gene Ther 6(12):1553-1560, 1995.

Muldoon LL, Nilaver G, Kroll RA, Pagel MA, Breakefield XO, Chiocca EA, Davidson BL, Weissleder R, Neuwelt EA.  Comparison of intracerebral osmotic blood-brain barrier disruption for delivery of adenovirus, herpesvirus, and iron oxide particles to normal rat brain.  Am J Pathol 147(6):1840-1851, 1995

Rios CD, Ooboshi H, Piegors D, Davidson BL, Heistad DD.  Adenovirus-mediated gene transfer to normal and atherosclerotic arteries: A novel approach.  ATVB 15(12):2241-2245, 1995.


Davidson BL, Doran SE, Shewach DS, Latta JM, Hartman JW, Roessler BJ.  Expression of Escherichia coli beta-galactosidase and rat HPRT in the CNS of Macaca mulatta following adenoviral mediated gene transfer.  Exp Neurol 125(2):258-267, 1994.

Davidson BL, Golovoy N, Roessler BJ.  A 13 base pair deletion in exon 1 of HPRTIllinois forms a functional GUG initiation codon.  Hum Genet 93(3):300-304, 1994.

Roessler BJ, Davidson BL.  Direct plasmid mediated transfection of adult murine brain cells in vivo using cationic liposomes.  Neurosci Lett 167(1-2):5-10, 1994. (Citations: 64)

Li T, Adamian M, Roof DJ, Berson EL, Dryja TP, Roessler BJ, Davidson BL.  In vivo transfer of a reporter gene to the retina mediated by an adenoviral vector.  Invest Ophthalmol Vis Sci 35(5):2543-2549, 1994.

Smythe WR, Hwang HC, Amin KM, Eck SL, Davidson BL, Wilson JM, Kaiser LR, Albelda SM.  Use of recombinant adenovirus to transfer the herpes simplex virus thymidine kinase (HSVtk) gene to thoracic neoplasms:  An effective in vitro drug sensitization system.  Cancer Res 54(8):2055-2059, 1994.

Shewach DS, Zerbe LK, Hughes TL, Roessler BJ, Breakefield XO, Davidson BL.  Enhanced cytotoxicity of antiviral drugs mediated by adenoviral directed transfer of the herpes simplex virus thymidine kinase gene in rat glioma cells.  Cancer Gene Ther 1(2):107-112, 1994.


Davidson BL, Brown JE, Weber CH, Palella TD, Roessler BJ.  Synthesis of normal and variant human hypoxanthine-guanine phosphoribosyltransferase in Escherichia coli.  Gene 123(2):271-275, 1993. (Citations: 2)

Davidson BL, Allen ED, Kozarsky KF, Wilson JM, Roessler BJ.  A model system for In vivo gene transfer to the central nervous system using an adenoviral vector.  Nat Genet 3(3):219-223, 1993. Highlighted in a commentary by Dr. X. Breakefield.

Roessler BJ, Allen ED, Wilson JM, Hartman JW, Davidson BL.  Adenoviral-mediated gene transfer to rabbit synovium in vivo.  J Clin Invest 92:1085-1092, 1993.


Davidson BL, Tarle SA, Van Antwerp ME, Gibbs DA, Watts RE, Kelley WN, Palella TD.  Identification of seventeen independent mutations responsible for human hypoxanthine-guanine phosphoribosyltransferase (HPRT) deficiency.  Am J Hum Genet 48(5):951-958, 1991.

Tarle SA, Davidson BL, Wu VC, Zidar FJ, Seegmiller JE, Kelley WN, Palella TD.  Determination of the mutations responsible for the Lesch-Nyhan syndrome in seventeen subjects.  Genomics 10:499-501, 1991.

Davidson BL, Roessler BJ, Palella TD.  Expression of normal and variant human hypoxanthine-guanine phosphoribosyltransferase in E. coli.  Adv Exp Med Biol 309B:105-108, 1991.


Davidson BL, Pashmforoush M, Kelley WN, Palella TD. Human hypoxanthine-guanine phosphoribosyltransferase deficiency:  The molecular defect in a patient with gout (HPRTAshville).  J Biol Chem 264:520-525, 1989.

Fujimori S, Davidson BL, Kelley WN, Palella TD.  Identification of a single nucleotide change in the hypoxanthine-guanine phosphoribosyltransferase gene (HPRTYale) responsible for Lesch-Nyhan syndrome.  J Clin Invest 83(1):11-13, 1989.

Davidson BL, Tarle SA, Palella TD, Kelley WN.  Molecular basis of hypoxanthine-guanine phosphoribosyltransferase (HPRT) deficiency in ten subjects determined by direct sequencing of amplified transcripts.  J Clin Invest 84(1)::342-346, 1989.


Davidson BL.  The genetic basis of hypoxanthine-guanine phosphoribosyltransferase deficiency states in humans.  Doctoral dissertation, 1988.

Davidson BL, Pashmforoush M, Kelley WN, Palella TD.  Genetic basis of hypoxanthine-guanine phosphoribosyltransferase deficiency in a patient with the Lesch-Nyhan syndrome (HPRTFlint).  Gene 63:331-336, 1988.

Davidson BL, Palella TD, Kelley WN.  Human hypoxanthine guanine phosphoribosyltransferase:  A single nucleotide substitution in cDNA clones isolated from a patient with Lesch-Nyhan syndrome (HPRTMidland).  Gene 68:85-91, 1988.

Fujimori S, Hidaka Y, Davidson BL, Palella TD, Kelley WN.  Identification of a single nucleotide change in a mutant for hypoxanthine-guanine phosphoribosyltransferase gene (HPRTAnn Arbor).  Hum Genet 79:39-43, 1988.

Davidson BL, Chen S-J, Wilson JM, Kelley WN, Palella TD.  Hypoxanthine-guanine phosphoribosyltransferase.  Genetic evidence for identical mutations in two partially deficient subjects.  J Clin Invest 82(6):2164-2167, 1988.


Wilson JM, Stout JT, Palella TD, Davidson BL, Kelley WN, Caskey CT.  A molecular survey of hypoxanthine-guanine phosphoribosyltransferase deficiency in man.  J Clin Invest 77(1):188-195, 1986.

Jensen SG, Davidson BL, Seip L.  Size variation among proteins induced by sugarcane mosaic viruses in plant tissues.  Phytopath 76:528-532, 1986.


Daddona PE, Davidson BL, Perignon JL, Kelley WN.  Genetic expression in partial adenosine deaminase deficiency mRNA levels and protein turnover for the enzyme variants in human B-lymphoblast cell lines.  J Biol Chem 260(6)3875-3880, 1985.


Daddona PE, Mitchell BS, Meuwissen HJ, Davidson BL, Wilson JM, Koller CA.  Adenosine deaminase deficiency with normal immune function.  An acidic mutation.  J Clin Invest  72:483-492, 1983.


Davidson BL, Boernke WE.  Amphibian ornithine aminotransferase:  some general biological and chemical characteristics.  Comp Biochem Physiol  72B:469-471, 1982.




Boudreau RL, Davidson BL.  RNAi therapy for neurodegenerative diseases – Chapter 3 in Curr Top Dev Biol 75:73-92, 2006


Harper SQ, Davidson BL.  Plasmid based RNA interference.  In: “RNA silencing: methods and protocols”, Methods in Molecular Biology, Chapter 11, 221-237, 2005.


Davidson BL. Viral delivery of recombinant shRNAs. In: Methods of Enzymology, Rossi J and Engelke D, eds. Elsevier Press, CA, In Press, 2004


Davidson BL.  Generating adenoviruses for shRNA or siRNA delivery.  In RNA Interference: Nuts and Bolts, Chapter 10, 209-219, 2003.

Chiorini JA, Davidson BL.  Recombinant adeno-associated virus vector types 4 and 5: preparation and application for CNS gene transfer. Methods in Molecular Medicine, Chapter 14, 269-285, 2003.


Davidson BL, Roessler BJ.  “Adenoviral mediated gene transfer: Potential therapeutic applications”.  In:  Viral Vectors:  Tools for the Study and Genetic Manipulation of the Nervous System, Kaplitt M, ed.  Academic Press, New York:  1995.


Davidson BL, Roessler BJ.  “Genetic basis of hypoxanthine-guanine phosphoribosyltrans-ferase (HPRT) deficiency.”  In:  Molecular Genetics, Biochemistry and Clinical Aspects of Inherited Disorders of Purine and Pyrimidine Metabolism, Gresser U, Zollner N, eds.  Berlin:  Springer-Verlag, Vol. 1A, Ch. 5, pp. 33-40, Aug. 1993.


Davidson BL, Palella TD, Kelley WN.  Hypoxanthine-guanine phosphoribosyltransferase deficiency:  Molecular basis and clinical relevance.  In: Genetic and Therapeutic Aspects of Lipid and Purine Metabolism, Wolfram G, ed.  Berlin:  Springer-Verlag, pp. 57-66, 1989.

Awards and Honors

2015, The Leslie Gehry Brenner Prize for Innovation in Science
2013, University of Iowa, Inventor Award
2012, University of Iowa, Innovator Award
2012, Carver College of Medicine Faculty Service Award
2011, Finalist, Pioneer Hi-Bred Iowa Women of Innovation Awards, Technology Association of Iowa
2011, S.J. DeArmond Lecture, American Association of Neuropathologists
2011, Distinguished Alumni Lecture, University of Michigan
2011, Presidential Lecture, University of Iowa
2010, Ramsey Lectureship, Iowa State University
2009, Mathilde Solowey Award, National Institutes of Health
2008, Eureka Award, National Institutes of Health
2007-2016, Carver Research Program of Excellence, University of Iowa
2007, Regents Faculty Excellence Award
2005, Monroe J Lustabder Memorial lecturer, Rutgers University
1999-2014, Roy J Carver Biomedical Research Chair
1990-1993, National Arthritis Foundation Postdoctoral Fellow Award
1988-1990, Fellowship for Protein Structure and Design, University of Michigan
1988, VI International Symposium on Human Purine and Pyrimidine Metabolism Scholarship Award
1980, Phi Kappa Phi Honor Society, Nebraska Wesleyan University
1979, Beta Beta Beta Biology Honorary Society, Nebraska Wesleyan University
1977-1981, Presidents Scholarship Award, Nebraska Wesleyan University

Editorial and Academic Positions

Editorial Positions

2013-present, Deputy Editor, Molecular Therapy
2007-present, Editorial Board, Human Molecular Genetics

Ad hoc reviewer

Science Translational Medicine
Nature Medicine
Nature Genetics
Nature Structural Mol Biol
Nature Communications
Am J Human Genetics
J Biol Chem
Nat Rev Neuroscience
Nat Rev Genetics
Nucleic Acids Research
Molecular Therapy
Gene Therapy
Human Gene Therapy
PLOS Genetics
PLOS Medicine

Academic and Institutional Committees

2017-present, Department of Pathology and Laboratory Medicine Committee on Appointments and Promotions
2017-2018, Search Committee Advisory Group, UPENN
2017-present, Translational Neuroscience Center Scientific Advisory Board, UPENN
2017-present, Neuroscience Advisory Committee, Mahoney Institute for Neuroscience (MINS), UPENN
2017-present, Department Committee on Appointments and Promotions, Path and Lab Medicine, UPENN
2017-present, Standing Chairs and Directors of Centers (SCDC), UPENN
2016-present, Dean's Advisory Council, UPENN
2016-present, Chair, Animal Space Advisory Committee, CHOP
2016-present, Chair, Research Space Committee, CHOP
2016-2019, Co-Chair, Research Information Services Advisory Committee (RISAC), CHOP
2016-2019, Committee on Appointments and Promotions, UPENN
2016-present, Understanding the Causes and Develop Therapies for Rare & Complex Diseases Work Group; Co-Chair, Genetics of Rare & Orphan Diseases Subgroup
2016-present, Chief Scientific Strategy Officer
2015-present, Research Advisory Council
2014-present, Neuroscience graduate program, UPENN
2014-present, CAMB graduate program, UPENN
2017-present, Scientific Advisory Board, Anhui IsoTex Biotech, Inc
2017-present, Packard Center Board of Advisors, Johns Hopkins University
2017-present, Scientific Advisory Board, STXBP1 Foundation
2017-present, International Selection Committee, 2018 Bower Award and Prize for Achievement in Science, Franklin Institute Awards
2016-present, Scientific Advisory Board, Friends of Telethon Italia
2016-present, Scientific Advisory Board, Intellia Therapeutics
2016-present, Scientific Director Search Committee, NIH/NINDS
2016-present, Chief Scientific Strategy Officer, Children's Hospital of Philadelphia Research Institute
2015-present, Scientific Advisory Board, Sarepta Therapeutics
2015-present, Advisory Board, Horizon 2020 Twinning 2015, European Commission
2015-present, Medical Research Advisory Board (MRAB), National Ataxia Foundation
2015-present, Data Safety Monitoring Board, Intrathecal Administration of scAAV9/JeT-GAN for the Treatment of Giant Axonal Neuropathy, sponsored by NIH
2013-present, Scientific Advisory Board, TIGEM, Naples, Italy
2012-present, Editorial Board of EMBO reports
2012-present, Scientific Advisory Board, nLIFE
2011-present, TAGS Study Section, NIH
2011-present, BDSRA Scientific Advisory Board
2009-present, CNS Therapy Advisory Board, UMass
2008-present, Scientific Advisory Board, NCL Foundation, Hamburg, Germany
2008-present, Scientific Advisory Board, Oregon National Primate Research Center
2007-present, Scientific Advisory Board, Robert Packard Center for ALS Research at Johns Hopkins
2000-present, Batten Disease Support and Research Association, Scientific Advisory Board
1997-present, American Society for Gene and Cell Therapy
 - 2018-present, Program Committee
 - 2016-present, Chair, Advisory Council

Leadership and Memberships

Memberships in Professional Organizations

2015-present, American Society of Human Genetics
 - 2015-present, Social Issues Committee
2014-present, National Advisory Neurological Disorders and Stroke Council, NIH/NINDS
2007-present, American Association for the Advancement of Science
 - 2007-present, Fellow
 - 2013-present, Chair, Medical Sciences
2017-present, American Academy of Arts and Sciences