Broadening the indications for hematopoietic stem cell genetic therapies.
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.
PubMed Abstracts
PubMed Abstracts