Germline alteration by gene therapy: assessing and reducing the risks.
Developments in gene therapy are certain to lead to the treatment of an increasing variety of diseases, some of which will affect patients who might wish to have children following their gene therapy treatment. These circumstances raise the concern that germline integration of gene therapy vector DNA could occur. Although our current understanding of reproductive biology and of the biodistribution of gene therapy vectors administered to extragonadal sites indicate that this risk is low, animal experiments and clinical studies designed specifically to address this question are warranted; because of this risk, every gene therapy vector should be tested for its potential to integrate into germ cells and preimplantation embryos.
Gordon
J W
JW
Dept of Neurobiology, Mt. Sinai Medical Center, New York, NY 10029, USA. jgordon@smtplink.mssm.edu
D000818:Animals; D001755:Blastocyst / Q000502:physiology; D005260:Female; D005817:Genetic Counseling; D015316:Genetic Therapy / Q000009:adverse effects*; D005822:Genetic Vectors; D018095:Germ-Line Mutation; D006801:Humans; D008297:Male; D009865:Oocytes / Q000502:physiology; D018570:Risk Assessment; D013090:Spermatocytes / Q000502:physiology