title: T lymphocyte-directed gene therapy for ADA- SCID: initial trial results after 4 years.
abstract: In 1990, a clinical trial was started using retroviral-mediated transfer of the adenosine deaminase (ADA) gene into the T cells of two children with severe combined immunodeficiency (ADA- SCID). The number of blood T cells normalized as did many cellular and humoral immune responses. Gene treatment ended after 2 years, but integrated vector and ADA gene expression in T cells persisted. Although many components remain to be perfected, it is concluded here that gene therapy can be a safe and effective addition to treatment for some patients with this severe immunodeficiency disease.
authors.affiliation: National Center for Human Genome Research, National Institutes of Health (NIH), Bethesda, MD 20892, USA.
authors.forename: R M
authors.initials: RM
authors.lastname: Blaese
authors.forename: K W
authors.initials: KW
authors.lastname: Culver
authors.forename: A D
authors.initials: AD
authors.lastname: Miller
authors.forename: C S
authors.initials: CS
authors.lastname: Carter
authors.forename: T
authors.initials: T
authors.lastname: Fleisher
authors.forename: M
authors.initials: M
authors.lastname: Clerici
authors.forename: G
authors.initials: G
authors.lastname: Shearer
authors.forename: L
authors.initials: L
authors.lastname: Chang
authors.forename: Y
authors.initials: Y
authors.lastname: Chiang
authors.forename: P
authors.initials: P
authors.lastname: Tolstoshev
authors.forename: J J
authors.initials: JJ
authors.lastname: Greenblatt
authors.forename: S A
authors.initials: SA
authors.lastname: Rosenberg
authors.forename: H
authors.initials: H
authors.lastname: Klein
authors.forename: M
authors.initials: M
authors.lastname: Berger
authors.forename: C A
authors.initials: CA
authors.lastname: Mullen
authors.forename: W J
authors.initials: WJ
authors.lastname: Ramsey
authors.forename: L
authors.initials: L
authors.lastname: Muul
authors.forename: R A
authors.initials: RA
authors.lastname: Morgan
authors.forename: W F
authors.initials: WF
authors.lastname: Anderson
mesh_terms: D000243:Adenosine Deaminase / Q000008:administration & dosage / Q000097:blood / Q000172:deficiency* / Q000235:genetics* / Q000627:therapeutic use; D000917:Antibody Formation; D001483:Base Sequence; D002648:Child; D002675:Child, Preschool; D005260:Female; D005500:Follow-Up Studies; D015870:Gene Expression; D018014:Gene Transfer Techniques; D015316:Genetic Therapy; D005822:Genetic Vectors; D006801:Humans; D007111:Immunity, Cellular; D018655:Lymphocyte Count; D017710:Lymphocyte Transfusion; D008214:Lymphocytes / Q000201:enzymology; D008969:Molecular Sequence Data; D016511:Severe Combined Immunodeficiency / Q000201:enzymology / Q000276:immunology / Q000628:therapy*; D013601:T-Lymphocytes / Q000201:enzymology / Q000276:immunology
chemical_list: D000243:Adenosine Deaminase; C051409:pegademase bovine