The potential for therapy of immune disorders with gene therapy.
Several hurdles remain before gene therapy will be a part of mainstream medical therapy; however, the preliminary report of success in HSC correction in patients with XSCID provides hope that gene therapy will become a reality.
Candotti
F
F
Disorders of Immunity Section, Clinical Gene Therapy Branch, National Human Genome Research Institute, National Institutes of Health, Bethesda, Maryland, USA.
D000818:Animals; D002675:Child, Preschool; D005260:Female; D015316:Genetic Therapy / Q000379:methods*; D006801:Humans; D007153:Immunologic Deficiency Syndromes / Q000175:diagnosis / Q000235:genetics* / Q000628:therapy*; D007223:Infant; D007231:Infant, Newborn; D008297:Male; D011379:Prognosis; D016896:Treatment Outcome