In Vivo Selection for Gene-Corrected HSPCs Advances Gene Therapy for a Rare Stem Cell Disease.
editorial · Level V
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- Record sourced from PubMed, PMID 31703769.
- Also identified by DOI 10.1016/j.stem.2019.10.004.
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Abstract
Two recent papers (one by Román-Rodríguez et al., 2019 in this issue of Cell Stem Cell) highlight how the power of biological selection on hematopoietic stem cell fitness can facilitate gene therapies for Fanconi Anemia. A clinical trial using lentiviral gene replacement and a proof-of-concept targeted genome editing study show robust engraftment and expansion of gene-corrected cells at levels reaching therapeutic relevance.
Medical subject headings
- Fanconi Anemia
- Hematopoietic Stem Cell Transplantation