Human gene therapy.
Somatic cell gene therapy for the correction of many human genetic diseases is now technically possible. We review several methods of gene transfer that have been successfully used in animal studies, and discuss the promise and potential limitations of these methods in the treatment of human genetic diseases.
Verma
I M
IM
Molecular Biology and Virology Laboratory, Salk Institute, San Diego, California 92816-5800.
Naviaux
R K
RK
D002478:Cells, Cultured / Q000637:transplantation; D030342:Genetic Diseases, Inborn / Q000453:epidemiology / Q000235:genetics / Q000628:therapy; D015316:Genetic Therapy / Q000379:methods*; D005822:Genetic Vectors; D006801:Humans; D015994:Incidence; D009928:Organ Specificity; D012190:Retroviridae