Precise correction of a spectrum of β-thalassemia mutations in coding and non-coding regions by base editors

• Main Authors: Kirti Prasad (Author), Nivedhitha Devaraju (Author), Anila George (Author), Nithin Sam Ravi (Author), Joshua Paul (Author), Gokulnath Mahalingam (Author), Vignesh Rajendiran (Author), Lokesh Panigrahi (Author), Vigneshwaran Venkatesan (Author), Kartik Lakhotiya (Author), Yogapriya Periyasami (Author), Aswin Anand Pai (Author), Yukio Nakamura (Author), Ryo Kurita (Author), Poonkuzhali Balasubramanian (Author), Saravanabhavan Thangavel (Author), Shaji R. Velayudhan (Author), Gregory A. Newby (Author), Srujan Marepally (Author), Alok Srivastava (Author), Kumarasamypet M. Mohankumar (Author)
• Format: Bog
• Udgivet: Elsevier, 2024-06-01T00:00:00Z.
• Fag: article
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β-thalassemia/HbE results from mutations in the β-globin locus that impede the production of functional adult hemoglobin. Base editors (BEs) could facilitate the correction of the point mutations with minimal or no indel creation, but its efficiency and bystander editing for the correction of β-thal...