ReviewMolecular therapy : the journal of the American Society of Gene Therapy2025
Treating genetic blood disorders in the era of CRISPR-mediated genome editing.
Review in Molecular therapy : the journal of the American Society of Gene Therapy, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 6 papers.
What it found
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
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Who cites it
6 citing papers in PubMed.
- CRISPR application in hematological disorders: from bench to bedside.Blood advances · 2026Review
- Review
- Review
- Clinical Significance of IKBKB Mutations:Saudi medical journal · 2026Review
- Advances in CRISPR/Cas9-Based Gene Editing in Filamentous Fungi.Journal of fungi (Basel, Switzerland) · 2025Review
- Precision Genome Engineering in Human Disease: Expanding Therapeutic Roles of CRISPR Technologies.Nigerian medical journal : journal of the Nigeria Medical AssociationArticle
Corrections and comments
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Authors and funding
5 authors.
Funding
No grant is acknowledged in the PubMed record.
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.
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Registered trials
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