Dr. Wilson is an experienced molecular biologist and a co-inventor of the first shRNA-expressing libraries. These libraries have been used in a pooled approach to identify and optimize shRNA sequences that induce selected phenotypes (as shRNAs or as siRNAs) in cell-culture models. Dr. Wilson also has experience in more conventional drug development, specifically for Friedreich Ataxia (FA).
Dr. Wilson is a practicing, board-certified molecular genetic pathologist with more than 20 years’ experience.
During a sabbatical in 2012-2013, Dr. Wilson studied advanced computational statistics at the University of Pennsylvania, culminating in graduate-level courses in Machine Learning and Advanced Bayesian Data Analysis.
Education and Training
MD - University of Pennsylvania, Philadelphia, PA
Residency in Clinical Pathology - Hospital of the University of Pennsylvania, Philadelphia, PA
Fellowship in Transfusion Medicine - Hospital of the University of Pennsylvania, Philadelphia, PA
Post-doctoral Research - Howard Hughes Medical Institute, University of Pennsylvania, Philadelphia, PA
American Board of Pathology/Molecular GeneticsClinical Pathology
PhD in Genetics - University of Pennsylvania, Philadelphia, PA
Titles and Academic Titles
Co-Director, Friedreich's Ataxia Center of Excellence
Professor of Pathology and Laboratory Medicine, Perelman School of Medicine at the University of Pennsylvania
Professor of Pathology and Laboratory Medicine in Pediatrics, Perelman School of Medicine at the University of Pennsylvania
Rufini A, Wilson RB, Testi R. Drug Discovery and Emerging Therapeutic Strategies. Journal of Rare Diseases. 2016;4(1):19-23.
Cotticelli MG, Acquaviva F, Xia S, Kaur A, Wang Y, Wilson RB. Phenotypic Screening for Friedreich Ataxia Using Random shRNA Selection. J Biomol Screen. 2015 Oct;20(9):1084-90.
Seyer L, Greeley N, Foerster D, Strawser C, Gelbard S, Dong Y, Schadt K, Cotticelli MG, Brocht A, Farmer J, Wilson RB, Lynch DR. Open label pilot study of interferon gamma-1b in Friedreich ataxia. Acta Neurol Scand. 2015 Jul;132(1):7-15.
Lazaropolous M, Dong Y, Clark E, Greeley NR, Seyer LA, Brigatti KW, Christie C, Perlman SL, Wilmot GR, Gomez CM, Mathews KD, Yoon G, Zesiewicz T, Hoyle C, Subramony SH, Brocht AF, Farmer JM, Wilson RB, Deutsch EC, Lynch DR. Frataxin levels in peripheral tissue in Friedreich ataxia. Ann Clin Transl Neurol. 2015 Aug;2(8):831-42.
Cotticelli MG, Acquaviva F, Xia S, Kaur A, Wang Y, Wilson RB. Phenotypic screening for Friedreich ataxia using random shRNA selection. J Biomol Screen. 2015 Oct;20(9):1084-90
Wang Y, Speier JS, Engram-Pearl J, Wilson RB. Introduction of mismatches in a random shRNA-encoding library improves potency for phenotypic selection. PLoS One. 2014;9(2):e87390.
Ogino, S. and Wilson, R.B.: Bayesian analysis. Molecular Pathology in Clinical Practice. Leonard, D.G., et al. (eds.). Springer, 2007.
Wilson, R.B.: Experimental Therapeutics for Friedreich Ataxia. Genetic Instabilities and Neurological Diseases. Wells, R.D. and Ashizawa, T. (eds.). Elsevier-Academic Press, 2006
Wilson, R.B.: Iron Dysregulation in Friedreich Ataxia. Seminars in Pediatric Neurology: Disorders of Brain Iron. Hayflick, S.J. and Bodensteiner, J.B. (eds.). Elsevier-Academic Press, 2006.
Posters and Presentations
Lectures by Invitation
Wilson RB. "Phenotypic drug screening using random shRNA selection." Sixth International Ataxia Investigators Meeting, National Ataxia Foundation, Orlando, FL, April 2016
Wilson RB. "p38 MAPK inhibitors for the Treatment of Friedreich's Ataxia." GlaxoSmithKline, King of Prussia, PA, March 2016
Wilson RB. "Friedreich's Ataxia Drug Discovery and Treatment Pipeline." Eighth Annual Children's Hospital of Philadelphia Friedreich's Ataxia Symposium, King of Prussia, PA, October 2015
Wilson RB. "Random shRNA Selection." New York Genome Center, New York, NY, September 2015
Wilson RB. "Therapeutic Discovery Using Random shRNA Selection." Columbia University College of Physicians and Surgeons, New York, NY, August 2015
Wilson RB. "Drug Discovery for Friedreich's Ataxia." Seventh Annual Children's Hospital of Philadelphia Friedreich's Ataxia Symposium, King of Prussia, PA, October 2014
Awards and Honors
2016, Top Doctor, Philadelphia, Castle Connolly
2013, Harrington Discovery Institute Scholar-Innovator Award
2011, 2011 Kevin E. Salhany Award for Excellence in Clinical Teaching, Department of Pathology and Laboratory Medicine, Hospital of the University of Pennsylvania2010, Distinguished Investigator Award, Friedreich's Ataxia Research Alliance
2009, NIH Director's Transformative Research Award
2009, Best Small Group Leader Teaching Award, University of Pennsylvania School of Medicine Class of 2012
2005, Basic Science Teaching Award, University of Pennsylvania School of Medicine Class of 2008
2004, Leonard Berwick Memorial Teaching Award, University of Pennsylvania School of Medicine
2003, Peter C. Nowell Teaching Award, Department of Pathology and Laboratory Medicine, Hospital of the University of Pennsylvania
2002, 2002 Kevin E. Salhany Award for Excellence in Clinical Teaching, Department of Pathology and Laboratory Medicine, Hospital of the University of Pennsylvania
2002, John Morgan Society, University of Pennsylvania
2001, The Outstanding Group Discussion Leader Teaching Award, University of Pennsylvania School of Medicine Class of 2003
1992, Young Investigator Award, Academy of Clinical Laboratory Physicians and Scientists
1989, Roy G. Williams Research Award, University of Pennsylvania School of Medicine
1982, Sigma Xi, Brown University
1982, Brand Musical Premium, Brown University
1982, Magna Cum Laude, Brown University
1982, Honors in Biochemistry, Brown University
1982, Honors in Music, Brown University
1980, Phi Beta Kappa, Brown University
Editorial and Academic Positions
1997-present, Ad Hoc Reviewer for Nature Genetics, Science, Neurology, Journal of Human Molecular Genetics, American Journal of Medical Genetics, European Journal of Human Genetics, Journal of Molecular Diagnostics, Leukemia and Lymphoma
2013-present, Committee on Appointments and Promotions, Perelman School of Medicine at the University of Pennsylvania
2010-present, Academic Appointments and Promotions Committee, Department of Pathology and Laboratory Medicine
2001-present, Selection Committees for Residents, Fellows, and Faculty, Department of Pathology and Laboratory Medicine
1998-present, Thesis Committees, Cell and Molecular Biology and Neuroscience Graduate Groups
Leadership and Memberships
2005-present, Friedreich's Ataxia Research Alliance (Scientific advisor)
1997-present, Association for Molecular Pathology (Clinical Practices Committee, 1997)
2002-present, National Ataxia Foundation (Medical Research Advisory Board)
Patient Experience Ratings
About the Patient Experience Rating System
The Patient Experience Rating is an average of all responses to the care provider related questions shown above from our nationally-recognized Press Ganey Patient Satisfaction Survey. Patients that are treated in outpatient or hospital environments may receive different surveys, and the volume of responses will vary by question.
Responses are measured on a scale of 1 to 5 with 5 being the best score.
We are committed to true transparency. However, to ensure the comments are fair and correctly attributed, we review each one before posting to the website. We exclude entire comments that disclose patient's protected health information, are off-topic, or include other confidential or inappropriate content. Comments will appear on provider bios only if providers have a minimum number of comments.
Comments are shared internally for education purposes to ensure that we are doing our very best for the patients and families for whom we are privileged to care.
The comments are submitted by patients and families and reflect their views and opinions. The comments are not endorsed by and do not reflect the views of Children’s Hospital of Philadelphia.