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Article

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

Cell stem cell - 7 Nov 2019

Gentner Bernhard, Naldini Luigi

Abstract excerpt

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...

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Identifiers

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

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026. Manifest publication-graph-cohort-2026-09-08.1.

Source receipt: pubmed, retrieved 7 Sept 2026.

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In Vivo Selection for Gene-Corrected HSPCs Advances Gene Therapy for a Rare Stem Cell Disease.PMID 31703769Read ↗

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Advances in Gene Therapy for Fanconi Anemia.Semantic proximity Read ↗

Survival of the fittest: in vivo selection and stem cell gene therapy.Semantic proximity Read ↗

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Stem Cell Collection and Gene Transfer in Fanconi AnemiaSemantic proximity Read ↗

Gene therapy: In vivo selection of gene-corrected cells.Semantic proximity Read ↗

Gene transfer for the eventual treatment of Fanconi's anemia.Semantic proximity Read ↗

In vivo selection using a cell-growth switch.Semantic proximity Read ↗

Gene therapy of Fanconi anemia: preclinical efficacy using lentiviral vectors.Semantic proximity Read ↗

Phenotype correction of Fanconi anemia group A hematopoietic stem cells using lentiviral vector.Semantic proximity Read ↗

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