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Article

Editing outside the body: Ex vivo gene-modification for β-hemoglobinopathy cellular therapy.

Molecular therapy : the journal of the American Society of Gene Therapy - 3 Nov 2021

Rosanwo Tolulope O, Bauer Daniel E

Abstract excerpt

Genome editing produces genetic modifications in somatic cells, offering novel curative possibilities for sickle cell disease and β-thalassemia. These opportunities leverage clinical knowledge of hematopoietic stem cell transplant and gene transfer. Advantages to this mode of ex vivo therapy include locus-specific alteration of patient hematopoietic stem cell genomes, lack of allogeneic immune response, and...

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Share to Anemia, Sickle Cell
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Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

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Source

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Identifiers

PMID: 34628053
DOI: 10.1016/j.ymthe.2021.10.002
PMCID: PMC8571174

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026. Manifest publication-graph-cohort-2026-09-07.1.

Source receipt: pubmed, retrieved 7 Sept 2026.

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