Steps toward gene therapy: 1. The initial trials.
Hopes for correcting the instruction tape in patients with genetic disease are being intensively explored. Initial efforts centered on monogenic immunodeficiency states, but in a pattern not wholly expected by researchers, the "acquired genetic diseases" of cancer and AIDS account for most ongoing clinical trials. Experience with adenosine deaminase deficiency shows the difficulties encountered in creating practical gene therapies.
Blaese
R M
RM
Clinical Gene Therapy Branch, National Center for Human Genome Research, National Institutes of Health, Bethesda, Md., USA.
D000243:Adenosine Deaminase / Q000172:deficiency*; D002675:Child, Preschool; D005818:Genetic Engineering; D015316:Genetic Therapy / Q000379:methods*; D005822:Genetic Vectors; D006801:Humans; D008979:Moloney murine leukemia virus; D016511:Severe Combined Immunodeficiency / Q000201:enzymology / Q000235:genetics / Q000628:therapy*
D000243:Adenosine Deaminase