title: Stem cell therapy for sickle cell disease: transplantation and gene therapy.
abstract: HLA-identical sibling hematopoietic cell transplantation (HCT) for sickle cell disease (SCD) has a strong track record of efficacy and there is growing appreciation that its benefits exceed its risks in selected individuals. In contrast, the clinical utility of replacement gene therapy for sickle cell disease remains unproven. Its challenge is to ensure viral transduction into hematopoietic stem cells (HSCs) and to generate safe, stable, erythroid-specific replacement gene expression at a level that is sufficient to have a clinical effect. The clinical necessity for fulfilling all these criteria may make this genetic disorder among the most complex to treat successfully by gene therapy. But the experience of HCT for SCD has proven that eliminating the beta(S)-globin gene is curative when the transfer is stable. Thus replacement gene therapy for sickle cell disease remains a subject of intense interest and investigation.
authors.affiliation: Children's Hospital and Research Center - Oakland, 747 52nd Street, Oakland, CA 94609-1809, USA. mwalters@mail.cho.org
authors.forename: Mark C
authors.initials: MC
authors.lastname: Walters
mesh_terms: D000755:Anemia, Sickle Cell / Q000235:genetics / Q000601:surgery / Q000628:therapy*; D018572:Disease-Free Survival; D015316:Genetic Therapy / Q000009:adverse effects / Q000379:methods*; D005822:Genetic Vectors; D006084:Graft Rejection / Q000453:epidemiology / Q000517:prevention & control; D006680:HLA Antigens / Q000235:genetics; D018380:Hematopoietic Stem Cell Transplantation / Q000379:methods*; D006801:Humans; D016086:Lentivirus / Q000235:genetics; D035781:Siblings; D018183:Transplantation Chimera; D019172:Transplantation Conditioning / Q000379:methods
chemical_list: D006680:HLA Antigens