Gene therapy and reproductive medicine.
OBJECTIVE
To review the literature on the principles of gene therapy and its potential application in reproductive medicine.


METHODS
Literature review.


METHODS
Gene therapy involves transfer of genetic material to target cells using a delivery system, or vector. Attention has primarily focused on viral vectors. Significant problems remain to be overcome including low efficacy of gene transfer, the transient expression of some vectors, safety issues with modified adenoviruses and retroviruses, and ethical concerns. If these issues can be resolved, gene therapy will be applicable to an increasing spectrum of single and multiple gene disorders, as the Human Genome Project data are analyzed, and the genetic component of human disease becomes better understood. Gynecologic gene therapy has advanced to human clinical trials for ovarian carcinoma, and shows potential for the treatment of uterine leiomyomata. Obstetric applications of gene therapy, including fetal gene therapy, remain more distant goals.


CONCLUSIONS
Concerns about the safety of human gene therapy research are being actively addressed, and remarkable progress in improving DNA transfer has been made. The first treatment success for a genetic disease (severe combined immunodeficiency disease) has been achieved, and ongoing research efforts will eventually yield clinical applications in many spheres of reproductive medicine.
Stribley
John M
JM
Department of Obstetrics and Gynecology, Reproductive Sciences Program, University of Michigan, Ann Arbor, Michigan 48109, USA.
Rehman
Khurram S
KS
Niu
Hairong
H
Christman
Gregory M
GM
D000256:Adenoviridae / Q000235:genetics; D004274:DNA, Recombinant; D005260:Female; D005315:Fetal Diseases / Q000628:therapy; D015316:Genetic Therapy / Q000009:adverse effects / Q000379:methods; D005822:Genetic Vectors; D005831:Genital Diseases, Female / Q000628:therapy*; D005833:Genital Neoplasms, Female / Q000628:therapy; D006801:Humans; D011247:Pregnancy; D012098:Reproduction; D012190:Retroviridae / Q000235:genetics
D004274:DNA, Recombinant