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Comment
. 2019 Nov 7;25(5):592-593.
doi: 10.1016/j.stem.2019.10.004.

In Vivo Selection for Gene-Corrected HSPCs Advances Gene Therapy for a Rare Stem Cell Disease

Bernhard Gentner  1 , Luigi Naldini  2
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In Vivo Selection for Gene-Corrected HSPCs Advances Gene Therapy for a Rare Stem Cell Disease

Bernhard Gentner et al. Cell Stem Cell. .
Free article

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.

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