Gene therapy progress and prospects: gene therapy for severe combined immunodeficiency.
Severe combined immunodeficiencies have long been targeted as a group of disorders amenable to gene therapy because of their defined molecular biology and pathophysiology, and the prediction that corrected cells would have profound growth and survival advantage. Recently, several clinical studies have shown that conventional gene transfer technology can produce major beneficial therapeutic effects in these patients, but, as for all cellular and pharmacological treatment approaches, with a finite potential for toxicity.
Gaspar
H B
HB
Molecular Immunology Unit, Institute of Child Health, London, UK.
Howe
S
S
Thrasher
A J
AJ
D000243:Adenosine Deaminase / Q000172:deficiency; D018014:Gene Transfer Techniques; D015316:Genetic Therapy / Q000379:methods* / Q000639:trends; D006801:Humans; D016254:Mutagenesis, Insertional; D016511:Severe Combined Immunodeficiency / Q000628:therapy*
D000243:Adenosine Deaminase