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Article

Treating genetic blood disorders in the era of CRISPR-mediated genome editing.

Molecular therapy : the journal of the American Society of Gene Therapy - 4 Jun 2025

Almotiri Alhomidi, Abogosh Ahmed, Abdelfattah Ali, Alowaisy Dalya, Rodrigues Neil P

Abstract excerpt

In the setting of monogenic disease, advances made in genome editing technologies can, in principle, be deployed as a therapeutic strategy to precisely correct a specific gene mutation in an affected cell type and restore functionality. Using the β-hemoglobinopathies and hemophilia as exemplars, we review recent experimental breakthroughs using CRISPR-derived genome editing technology that have translated to...

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Identifiers

PMID: 39827371
DOI: 10.1016/j.ymthe.2025.01.031
PMCID: PMC12172200

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