Broadening the indications for hematopoietic stem cell genetic therapies.
editorial · Level V
Where this comes from
- Record sourced from PubMed, PMID 24012366.
- Also identified by DOI 10.1016/j.stem.2013.08.006.
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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.
Medical subject headings
- Cerebroside-Sulfatase
- Genetic Therapy
- Hematopoietic Stem Cell Transplantation
- Hematopoietic Stem Cells
- Leukodystrophy, Metachromatic
- Wiskott-Aldrich Syndrome
- Wiskott-Aldrich Syndrome Protein