title: Hematopoietic stem cell gene therapy.
abstract: Gene therapy applications that target hematopoietic stem cells (HSCs) offer great potential for the treatment of hematologic disease. Despite this promise, clinical success has been limited by poor rates of gene transfer, poor engraftment of modified cells, and poor levels of gene expression. We describe here the basic approach used for HSC gene therapy, briefly review some of the seminal clinical trials in the field, and describe several recent advances directed toward overcoming these limitations.
authors: University of Washington Department of Medicine, Seattle 98195-7720, USA.
authors: David W
authors: DW
authors: Emery
authors: Tamon
authors: T
authors: Nishino
authors: Ken
authors: K
authors: Murata
authors: Michalis
authors: M
authors: Fragkos
authors: George
authors: G
authors: Stamatoyannopoulos
mesh_terms: D000818:Animals; D018014:Gene Transfer Techniques; D015316:Genetic Therapy / Q000379:methods*; D005822:Genetic Vectors; D006402:Hematologic Diseases / Q000628:therapy*; D018380:Hematopoietic Stem Cell Transplantation / Q000379:methods*; D006801:Humans; D016086:Lentivirus / Q000235:genetics; D023421:Models, Animal; D016092:Spumavirus / Q000235:genetics