Prospects for fetal gene therapy.
Advances in prenatal diagnosis and gene transfer technology have allowed consideration of prenatal gene therapy. A compelling argument can be made for this strategy in treating genetic diseases that are fatal in the prenatal or perinatal period. In other diseases, the fetal environment may offer unique biological advantages that favor a prenatal gene therapy strategy over treatment after birth. Although issues of safety and efficacy must be resolved before clinical application, the development of fetal gene therapy may become a new molecular therapeutic arm in the field of prenatal intervention.
Yang
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EY
Department of Surgery, the Center for Fetal Diagnosis and Treatment, Children's Hospital of Philadelphia, PA 19104, USA.
Flake
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Adzick
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D005260:Female; D005315:Fetal Diseases / Q000628:therapy*; D005332:Fetoscopy; D015316:Genetic Therapy; D005822:Genetic Vectors; D006801:Humans; D008099:Liver / Q000201:enzymology; D008171:Lung Diseases / Q000628:therapy; D008661:Metabolism, Inborn Errors / Q000235:genetics / Q000628:therapy; D011247:Pregnancy