Prenatal gene therapy: can the technical hurdles be overcome?
Fifty years ago, a medical breakthrough in the prenatal diagnosis of genetic disorders was made with the introduction of amniocentesis. Until recently, there was little hope that diseases diagnosed in utero could be treated before birth. Today, prenatal gene therapy is emerging as a new concept for treating pre- and postnatal manifestations of genetic diseases and developmental disorders. Research studies have generated a degree of optimism by demonstrating the feasibility of fetal gene transfer. Nevertheless, enthusiasm is tempered by the considerable technical and ethical issues raised by such studies. Undoubtedly, the future of prenatal gene transfer as a therapeutic approach for birth defects mostly depends on addressing and overcoming these concerns.
Senut
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The Salk Institute for Biological Studies, Laboratory of Genetics, PO Box 85800, San Diego, CA 92186-5800, USA. senut@salk.edu
Gage
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D000818:Animals; D005260:Female; D005315:Fetal Diseases / Q000235:genetics* / Q000628:therapy*; D018014:Gene Transfer Techniques; D015316:Genetic Therapy / Q000379:methods*; D005822:Genetic Vectors; D006801:Humans; D010920:Placenta; D011247:Pregnancy; D011296:Prenatal Diagnosis; D012307:Risk Factors; D019076:Transgenes