The level of sophistication of prenatal imaging and genetic diagnostics is growing rapidly, however cures for many congenital anomalies lag behind our diagnostic abilities. In developmental defects where babies are born with a disease phenotype, fetal therapy offers an opportunity to prevent or cure these disease. This is an opportunity that is lost after birth.
There are 3 broad aims of my basic science laboratory: 1) In Utero gene editing for the treatment of genetic diseases, 2) In Utero reversal of lung hypoplasia in diaphragmatic hernia 3) Minimally invasive In Utero therapy for spina bifida.
- Life-Long Transgene Expression in Skeletal Muscle Without Transduction of Satellite Cells Following Embryonic Myogenic Progenitor Transduction by Lentivirus Administered in Utero. Stem Cells Dev. 2015 Aug 15;24(16):1878-87. doi: 10.1089/scd.2014.0585. Epub 2015 Jun 9.
- Developmental stage determines efficiency of gene transfer to muscle satellite cells by in utero delivery of adeno-associated virus vector serotype 2/9. Mol Ther Methods Clin Dev. 2014 Sep 10;1:14040. doi: 10.1038/mtm.2014.40. eCollection 2014.
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- Gene Ther. 2010 Jan;17(1):61-71. doi: 10.1038/gt.2009.115. Epub 2009 Sep 3.