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

• Main Authors: Anila George (Author), Poornasree Sadanandan (Author), Nithin Sam Ravi (Author), B. Vaishnavi (Author), Srujan Marepally (Author), Saravanbhavan Thangavel (Author), Shaji R. Velayudhan (Author), Alok Srivastava (Author), Kumarasamypet M Mohankumar (Author)
• Format: Book
• Published: Elsevier, 2024-12-01T00:00:00Z.
• Subjects: article
• Online Access: Connect to this object online.

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...