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

Almotiri, Alhomidi, Abogosh, Ahmed, Abdelfattah, Ali, Alowaisy, Dalya and Rodrigues, Neil P. ORCID: https://orcid.org/0000-0002-1925-7733 2025. Treating genetic blood disorders in the era of CRISPR-mediated genome editing. Molecular Therapy 10.1016/j.ymthe.2025.01.031 Item availability restricted.

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Abstract

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 significant improvements in the management of inherited hematologic disorders. Yet there are also challenges facing the use of CRISPR-mediated genome editing in these patients; we discuss possible ways to obviate those issues for furtherance of clinical benefit.

Item Type:	Article
Date Type:	Published Online
Status:	In Press
Schools:	Biosciences
Publisher:	Cell Press
ISSN:	1525-0016
Date of First Compliant Deposit:	16 January 2025
Date of Acceptance:	14 January 2025
Last Modified:	13 Feb 2025 09:30
URI:	https://orca.cardiff.ac.uk/id/eprint/175309

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