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

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...

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Principais autores: Kirti Prasad, Nivedhitha Devaraju, Anila George, Nithin Sam Ravi, Joshua Paul, Gokulnath Mahalingam, Vignesh Rajendiran, Lokesh Panigrahi, Vigneshwaran Venkatesan, Kartik Lakhotiya, Yogapriya Periyasami, Aswin Anand Pai, Yukio Nakamura, Ryo Kurita, Poonkuzhali Balasubramanian, Saravanabhavan Thangavel, Shaji R. Velayudhan, Gregory A. Newby, Srujan Marepally, Alok Srivastava, Kumarasamypet M. Mohankumar
Formato: Artigo
Idioma:Inglês
Publicado em: Elsevier 2024-06-01
coleção:Molecular Therapy: Nucleic Acids
Assuntos:
MT: RNA/DNA Editing
genome editing
base editor
β-thalassemia mutations
HbE/CD26(G>A)
gene correction
Acesso em linha:http://www.sciencedirect.com/science/article/pii/S2162253124000921
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