Hematopoietic stem cell gene therapy.
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.
Emery
David W
DW
University of Washington Department of Medicine, Seattle 98195-7720, USA.
Nishino
Tamon
T
Murata
Ken
K
Fragkos
Michalis
M
Stamatoyannopoulos
George
G
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