Fetal Gene Therapy Research

Our team of world-renowned experts are at the forefront of developing innovative treatments and therapies to solve underlying prenatally diagnosed conditions through stem cell and gene therapy. Through innovative research and discoveries, our team continues to make breakthroughs in research with game-changing success.

Current research

• Maternal Fetal Antibody Levels
• Amniotic Fluid Registry
• Prenatal Sickle Cell Disease Research
• Fetal Stem Cell Transplantation

Highlights

• Peranteau WH, Flake AW. The Future of In Utero Gene Therapy. Mol Diagn Ther. 2020 Apr;24(2):135-142.
• Palanki R, Peranteau WH, Mitchell MJ. Delivery technologies for in utero gene therapy. Adv Drug Deliv Rev. 2020 Nov 9;169:51-62.
• O'Connell AE, Guseh S, Lapteva L, Cummings CL, Wilkins-Haug L, Chan J, Peranteau WH, Almeida-Porada G, Kourembanas S. Gene and Stem Cell Therapies for Fetal Care: A Review. JAMA Pediatr. 2020 Oct 1;174(10):985-991.

Prior noteworthy publications

• Alapati D, Zacharias WJ, Hartman HA, Rossidis AC, Stratigis JD, Ahn NJ, Coons B, Zhou S, Li H, Singh K, Katzen J, Tomer Y, Chadwick AC, Musunuru K, Beers MF, Morrisey EE, Peranteau WH. In utero gene editing for monogenic lung disease. Sci Transl Med. 2019 Apr 17;11(488):eaav8375.
• Loukogeorgakis SP, Shangaris P, Bertin E, Franzin C, Piccoli M, Pozzobon M, Subramaniam S, Tedeschi A, Kim AG, Li H, Fachin CG, Dias AIBS, Stratigis JD, Ahn NJ, Thrasher AJ, Bonfanti P, Peranteau WH, David AL, Flake AW, De Coppi P. In Utero Transplantation of Expanded Autologous Amniotic Fluid Stem Cells Results in Long-Term Hematopoietic Engraftment. Stem Cells. 2019 Sep;37(9):1176-1188.
• Rossidis AC, Stratigis JD, Chadwick AC, Hartman HA, Ahn NJ, Li H, Singh K, Coons BE, Li L, Lv W, Zoltick PW, Alapati D, Zacharias W, Jain R, Morrisey EE, Musunuru K, Peranteau WH. In utero CRISPR-mediated therapeutic editing of metabolic genes. Nat Med. 2018 Oct;24(10):1513-1518.
• Ahn NJ, Stratigis JD, Coons BE, Flake AW, Nah-Cederquist HD, Peranteau WH. Intravenous and Intra-amniotic In Utero Transplantation in the Murine Model. J Vis Exp. 2018 Oct 9;(140):58047.
• Witt R, MacKenzie TC, Peranteau WH. Fetal stem cell and gene therapy. Semin Fetal Neonatal Med. 2017 Dec;22(6):410-414.
• McClain LE, Flake AW. In utero stem cell transplantation and gene therapy: Recent progress and the potential for clinical application. Best Pract Res Clin Obstet Gynaecol. 2016 Feb;31:88-98.
• Loukogeorgakis SP, Flake AW. In utero stem cell and gene therapy: current status and future perspectives. Eur J Pediatr Surg. 2014 Jun;24(3):237-45.
• Pearson EG, Flake AW. Stem cell and genetic therapies for the fetus. Semin Pediatr Surg. 2013 Feb;22(1):56-61.
• 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.
• Roybal JL, Santore MT, Flake AW. Stem cell and genetic therapies for the fetus. Semin Fetal Neonatal Med. 2010 Feb;15(1):46-51.
• Yang EY, Flake AW, Adzick NS. Prospects for fetal gene therapy. Semin Perinatol. 1999 Dec;23(6):524-34.
• Yang EY, Cass DL, Sylvester KG, Wilson JM, Adzick NS. BAPS Prize--1997. Fetal gene therapy: efficacy, toxicity, and immunologic effects of early gestation recombinant adenovirus. British Association of Paediatric Surgeons. J Pediatr Surg. 1999 Feb;34(2):235-41.