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

Bernhard Gentner; Luigi Naldini

doi:10.1016/j.stem.2019.10.004

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

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

Authors

Bernhard Gentner¹, Luigi Naldini²

Affiliations

¹ San Raffaele Telethon Institute for Gene Therapy, Milan, Italy; Hematology and Bone Marrow Transplantation Unit, IRCCS San Raffaele Scientific Institute, Milan, Italy. Electronic address: gentner.bernhard@hsr.it.
² San Raffaele Telethon Institute for Gene Therapy, Milan, Italy; Vita Salute San Raffaele University, Milan, Italy. Electronic address: naldini.luigi@hsr.it.

PMID: 31703769
DOI: 10.1016/j.stem.2019.10.004

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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MeSH terms

CRISPR-Cas Systems
DNA Breaks
Fanconi Anemia*
Genetic Therapy
Genetic Vectors
Hematopoietic Stem Cell Transplantation*
Humans
Lentivirus / genetics
Mutation