Gene therapy for metabolic disorders.
Most diseases caused by genetic deficiencies could, in theory, be treated by the introduction and expression of a normal gene into an appropriate target tissue. It seems likely that gene therapy strategies for most metabolic disorders will not require strict gene regulation, as a fraction of the normal levels of gene activity could result in amelioration or significant improvement in the clinical outcome. Gene therapy is making rapid progress towards the goal of treating various disorders: here, we summarize the state of gene therapy for metabolic disorders.
Kay
M A
MA
Markey Molecular Medicine Center, Department of Medicine, University of Washington, Seattle 98195.
Woo
S L
SL
D000818:Animals; D015316:Genetic Therapy; D005822:Genetic Vectors; D006801:Humans; D008659:Metabolic Diseases / Q000628:therapy*