Editing of homologous globin genes by nickase-deficient base editor mitigates large intergenic deletions in HSPCs

Editing of homologous globin genes by nickase-deficient base editor mitigates large intergenic deletions in HSPCs

Recent studies have shown that base editing, even with single-strand breaks, could result in large deletions of the interstitial regions while targeting homologous regions. Several therapeutically relevant genes such as HBG, HBB, CCR5, and CD33 have homologous sites and are prone for large deletion...

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Bibliographic Details
Main Authors: Anila George, Poornasree Sadanandan, Nithin Sam Ravi, B. Vaishnavi, Srujan Marepally, Saravanbhavan Thangavel, Shaji R. Velayudhan, Alok Srivastava, Kumarasamypet M Mohankumar
Format: Article
Language:English
Published: Elsevier 2024-12-01
Series:Molecular Therapy: Nucleic Acids
Subjects:
MT: RNA/DNA Editing
base editing
CRISPR-Cas9
gamma globin
hemoglobinopathies
large deletions
Online Access:http://www.sciencedirect.com/science/article/pii/S2162253124002348

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http://www.sciencedirect.com/science/article/pii/S2162253124002348

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