Fetal Gene Therapy Publications and Information | The Children's Hospital of Philadelphia

Center for Fetal Diagnosis and Treatment

Fetal Discussion Forum

Talk with other families about fetal diagnoses.

Visit the Fetal Discussion Forums »

Fetal Gene Therapy Publications

The following list of Fetal Gene Therapy publications will help you in your search for more information. Most of our Fetal Gene Therapy publications are written by members of the Fetal Diagnosis and Treatment at The Children's Hospital of Philadelphia. If you are unable to obtain any of these Fetal Gene Therapy publications, please let us know, and we will send copies to you.

Journal articles

2012

Abi-Nader KN, Boyd M, Flake AW, Mehta V, Peebles D, David AL. Animal models for prenatal gene therapy: the sheep model. Methods Mol Biol. 2012;891:219-48. Cited in PubMed; PMID:22648775. Read the abstract

Roybal JL, Endo M, Buckley SM, Herbert BR, Waddington SN, Flake AW. Animal models for prenatal gene therapy: rodent models for prenatal gene therapy. Methods Mol Biol. 2012;891:201-18. Cited in PubMed; PMID:22648774. Read the abstract 

Laje P, Johnson MP, Howell LJ, Bebbington MW, Hedrick HL, Flake AW, et al. Ex utero intrapartum treatment in the management of giant cervical teratomas. J Pediatr Surg. 2012 Jun;47(6):1208-16. Cited in PubMed; PMID:22703795. Read the abstract 

2011

Davey MG, Flake AW. Genetic therapy for the fetus: a once in a lifetime opportunity. Hum Gene Ther. 2011 Apr;22(4):383-5. Cited in PubMed; PMID 21486158. No abstract available.

2010

Endo M, Henriques-Coelho T, Zoltick PW, Stitelman DH, Peranteau WH, Radu A, Flake AW. The developmental stage determines the distribution and duration of gene expression after early intra-amniotic gene transfer using lentiviral vectors. Gene Ther. 2010 Jan;17(1):61-71. Epub 2009 Sep 3.

2009

Laje P, Shang D, Cao W, Niiya M, Endo M, Radu A, DeRogatis N, Scheiflinger F, Zoltick PW, Flake AW, Zheng XL. Correction of murine ADAMTS13 deficiency by hematopoietic progenitor cell-mediated gene therapy. Blood. 2009 Mar 5;113(10):2172-80. Epub 2009 Jan 13.

Niiya M, Endo M, Shang D, Zoltick PW, Muvarak NE, Cao W, Jin SY, Skipwith CG, Motto DG, Flake AW, Zheng XL. Correction of ADAMTS13 deficiency by in utero gene transfer of lentiviral vector encoding ADAMTS13 genes. Mol Ther. 2009 Jan;17(1):34-41. Epub 2008 Oct 28.

2002

Hayashi S, Peranteau W, Shaaban AF, Flake AW. Complete allogeneic hematopoietic chimerism achieved by a combined strategy of in utero hematopoietic stem cell transplantation and postnatal donor lymphocyte infusion. Blood 100:804-812, 2002

MacKenzie TC, Kobinger GP, Kootstra NA, Radu A, Sena-Esteves M, Bouchard S,Wilson JM, Verma IM, Flake AW. Efficient transduction of liver and muscle after in utero injection of lentiviral vectors with different pseudotypes. Molecular Therapy 6:349-358, 2002

MacKenzie TC, Shaaban AF, Radu A, Flake AW. Engraftment of bone marrow and fetal liver cells after in utero transplantation in mdx mice. Journal of Pediatric Surgery 37:1058-1064, 2002

Peranteau WH, Hayashi S, Hsieh M, Shaaban AF, Flake AW. High level allogeneic chimerism achieved by a strategy of prenatal tolerance induction and postnatal non-myeloablative bone marrow transplantation. Blood. 2002 Sep 15;100(6):2225-34.

Krupnick AS, Shaaban AF, Flake AW. Bone marrow tissue engineering. Tissue Engineering 8:145-155, 2002.

Wilson RD. Cystic fibrosis carrier testing in pregnancy in Canada. JOGC 24:644-647, 2002.

2001

MacKenzie TC, Flake AW. Multilineage differentiation of human MSC after in utero transplantation. Cytotherapy 3:403-405, 2001

Arruda VR, Fields PA, Milner R, Wainwright L, De Miguel MP, Donovan PJ, Herzog RW, Nichols TC, Biegel JA, Razavi M, Dake M, Huff D, Flake AW, Couto L, Kay MA, High KA. Lack of germline transmission of vector sequences following systemic administration of recombinant AAV-2 vector in males. Molecular Therapy 6:586-592, 2001

2000

Kay MA, Manno CS, Ragni MV, Larson PJ, Couto LB, McClelland A, Glader B, Chew AJ, Tai SJ, Herzog RW, Arruda V, Johnson F, Scallan C, Skarsgard E, Flake AW, High KA: Evidence for gene transfer and expression of blood coagulation factor IX in patients with severe haemophilia B treated with an AAV vector. Nature Genetics 24:257-261, 2000.

1999

Yang EY, Kim HB, Shaaban AF, Milner R, Adzick NS, Flake AW. Fetal intramuscular delivery of recombinant adenovirus results in persistent postnatal transgene expression in both muscle and liver. J Pediatr Surg. 1999 May;34(5):766-72; discussion 772-3.

1997

Yang EY, Cass DL, Sylvester KG, Flake AW, Wilson JM, Adzick NS: Liver-directed fetal gene therapy using recombinant adenoviral vectors: Successful readministration in mid-gestation sheep fetuses. Surgical Forum, 47: 464-467, 1997.

Sylvester KG, Yang EY, Cass DL, Crombleholme TM, Adzick NS: Fetoscopic gene therapy for congenital lung disease. J Pediatr Surg 32:964-969, 1997.

1995

Meuli M, Liu Y, Liggit D, Meuli-Simmen C, Knauer S, Harrison MR, Adzick NS, Debs RJ: Cutaneous gene expression after local DNA delivery. Surgical Forum 46:701-702, 1995.

1987

Eglitis MA, Kantoff PW, McLachlin JR, Gillio A, Flake AW: Gene therapy: efforts at developing large animal models for autologous bone marrow transplant and gene transfer with retroviral vectors. Ciba Foundation Symposium 130:229-246 1987.

1986

Kantoff PW, Gillio A, McLachlin JR, Flake AW, Eglitis MA, Moen R, Karlsson S, Kohn DB, Karson E, Zweibel JA: Retroviral-mediated gene transfer into hematopoietic cells. Transactions of the Association of American Physicians 99:92-102, 1986.

Chapters

Evans MI, Flake AW, Harrison MR, Quintero RA, Johnson MP. Fetal Therapy. In Genetic Disorders and the Fetus, 5th edition, Milunsky A (ed.), The John Hopkins University Press, Baltimore, MD, Chapter 29, pp 952 98, 1998.

Johnson MP, Harrison MR, Adzick NS, Reichler A, Evans MI. Fetal gene therapy. In Rock J and Gant N (eds): Advances in Obstetrics and Gynecology. CV Mosby Publishing Company, St. Louis MO; 1994, pp 423-434.

Back to top »

Back to publications index »

  • Print
  • Share

Contact Us