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

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Title: Precise correction of a spectrum of β-thalassemia mutations in coding and non-coding regions by base editors.
Authors: Prasad K; Centre for Stem Cell Research (a Unit of inStem, Bengaluru), Christian Medical College Campus, Bagayam, Vellore, Tamil Nadu 632002, India.; Manipal Academy of Higher Education, Karnataka 576104, India., Devaraju N; Centre for Stem Cell Research (a Unit of inStem, Bengaluru), Christian Medical College Campus, Bagayam, Vellore, Tamil Nadu 632002, India.; Manipal Academy of Higher Education, Karnataka 576104, India., George A; Centre for Stem Cell Research (a Unit of inStem, Bengaluru), Christian Medical College Campus, Bagayam, Vellore, Tamil Nadu 632002, India.; Sree Chitra Tirunal Institute for Medical Sciences and Technology, Thiruvananthapuram, Kerala 695 011, India., Ravi NS; Centre for Stem Cell Research (a Unit of inStem, Bengaluru), Christian Medical College Campus, Bagayam, Vellore, Tamil Nadu 632002, India.; Sree Chitra Tirunal Institute for Medical Sciences and Technology, Thiruvananthapuram, Kerala 695 011, India., Paul J; Centre for Stem Cell Research (a Unit of inStem, Bengaluru), Christian Medical College Campus, Bagayam, Vellore, Tamil Nadu 632002, India.; Manipal Academy of Higher Education, Karnataka 576104, India., Mahalingam G; Centre for Stem Cell Research (a Unit of inStem, Bengaluru), Christian Medical College Campus, Bagayam, Vellore, Tamil Nadu 632002, India., Rajendiran V; Centre for Stem Cell Research (a Unit of inStem, Bengaluru), Christian Medical College Campus, Bagayam, Vellore, Tamil Nadu 632002, India.; Sree Chitra Tirunal Institute for Medical Sciences and Technology, Thiruvananthapuram, Kerala 695 011, India., Panigrahi L; Centre for Stem Cell Research (a Unit of inStem, Bengaluru), Christian Medical College Campus, Bagayam, Vellore, Tamil Nadu 632002, India.; Manipal Academy of Higher Education, Karnataka 576104, India., Venkatesan V; Centre for Stem Cell Research (a Unit of inStem, Bengaluru), Christian Medical College Campus, Bagayam, Vellore, Tamil Nadu 632002, India.; Manipal Academy of Higher Education, Karnataka 576104, India., Lakhotiya K; Molecular Cardiology Research Institute, Tufts Medical Center, 800 Washington Street, Boston MA 02111, USA., Periyasami Y; Centre for Stem Cell Research (a Unit of inStem, Bengaluru), Christian Medical College Campus, Bagayam, Vellore, Tamil Nadu 632002, India., Pai AA; Sree Chitra Tirunal Institute for Medical Sciences and Technology, Thiruvananthapuram, Kerala 695 011, India.; Department of Haematology, Christian Medical College & Hospital, Vellore 632 004, India., Nakamura Y; Cell Engineering Division, RIKEN BioResource Center, 3-1-1 Koyadai, Tsukuba, Ibaraki 3050074, Japan., Kurita R; Research and Development Department, Central Blood Institute Blood Service Headquarters, Japanese Red Cross Society, Tokyo, Japan., Balasubramanian P; Sree Chitra Tirunal Institute for Medical Sciences and Technology, Thiruvananthapuram, Kerala 695 011, India.; Department of Haematology, Christian Medical College & Hospital, Vellore 632 004, India., Thangavel S; Centre for Stem Cell Research (a Unit of inStem, Bengaluru), Christian Medical College Campus, Bagayam, Vellore, Tamil Nadu 632002, India., Velayudhan SR; Centre for Stem Cell Research (a Unit of inStem, Bengaluru), Christian Medical College Campus, Bagayam, Vellore, Tamil Nadu 632002, India.; Department of Haematology, Christian Medical College & Hospital, Vellore 632 004, India., Newby GA; Departments of Genetic Medicine and Biomedical Engineering, Johns Hopkins University School of Medicine, Baltimore, MD 21205, USA., Marepally S; Centre for Stem Cell Research (a Unit of inStem, Bengaluru), Christian Medical College Campus, Bagayam, Vellore, Tamil Nadu 632002, India., Srivastava A; Centre for Stem Cell Research (a Unit of inStem, Bengaluru), Christian Medical College Campus, Bagayam, Vellore, Tamil Nadu 632002, India.; Department of Haematology, Christian Medical College & Hospital, Vellore 632 004, India., Mohankumar KM; Centre for Stem Cell Research (a Unit of inStem, Bengaluru), Christian Medical College Campus, Bagayam, Vellore, Tamil Nadu 632002, India.; Manipal Academy of Higher Education, Karnataka 576104, India.
Source: Molecular therapy. Nucleic acids [Mol Ther Nucleic Acids] 2024 May 03; Vol. 35 (2), pp. 102205. Date of Electronic Publication: 2024 May 03 (Print Publication: 2024).
Publication Type: Journal Article
Journal Info: Publisher: Cell Press Country of Publication: United States NLM ID: 101581621 Publication Model: eCollection Cited Medium: Print ISSN: 2162-2531 (Print) Linking ISSN: 21622531 NLM ISO Abbreviation: Mol Ther Nucleic Acids Subsets: PubMed not MEDLINE
Database: MEDLINE Ultimate
Description
ISSN:2162-2531
DOI:10.1016/j.omtn.2024.102205