title: Broadening the indications for hematopoietic stem cell genetic therapies.
abstract: The use of recombinant retroviral vectors to effect corrective genetic therapies in hematopoietic stem cells (HSCs) has long been predicted to revolutionize medicine. Two recent papers in Science now show that this technology could be considered as effective as, and perhaps superior to, allogeneic HSC transplants in some rare diseases.
authors.affiliation: Division of Hematology/Oncology, Children's Hospital Boston, 300 Longwood Avenue, Boston, MA 02115, USA. dawilliams@childrens.harvard.edu
authors.forename: David A
authors.initials: DA
authors.lastname: Williams
mesh_terms: D002553:Cerebroside-Sulfatase / Q000235:genetics*; D015316:Genetic Therapy / Q000379:methods*; D018380:Hematopoietic Stem Cell Transplantation; D006412:Hematopoietic Stem Cells / Q000378:metabolism*; D006801:Humans; D007966:Leukodystrophy, Metachromatic / Q000628:therapy*; D008297:Male; D014923:Wiskott-Aldrich Syndrome / Q000628:therapy*; D051316:Wiskott-Aldrich Syndrome Protein / Q000235:genetics*
chemical_list: D051316:Wiskott-Aldrich Syndrome Protein; D002553:Cerebroside-Sulfatase