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Base editing of key residues in the BCL11A-XL-specific zinc finger domains derepresses fetal globin expression.

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Title: Base editing of key residues in the BCL11A-XL-specific zinc finger domains derepresses fetal globin expression.
Authors: 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., 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, Manipal, Karnataka 576104, India., Haddad M; School of Biotechnology and Biomolecular Sciences, University of New South Wales, Sydney, NSW, Australia., 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., 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, Manipal, Karnataka 576104, 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, Manipal, Karnataka 576104, India., Gopalakrishnan C; Department of Integrative Biology, School of Bioscience and Technology, Vellore Institute of Technology (VIT, Deemed to be University), Vellore, Tamil Nadu 632014, India., Wyman S; Innovative Genomics Institute, University of California, Berkeley, Berkeley, CA 94704, USA., Ariudainambi K; Department of Biochemistry, Auxilium College, Vellore, Tamil Nadu 632006, India., Mahalingam G; Centre for Stem Cell Research (a Unit of inStem, Bengaluru), Christian Medical College Campus, Bagayam, Vellore, Tamil Nadu 632002, India., Periyasami Y; Centre for Stem Cell Research (a Unit of inStem, Bengaluru), Christian Medical College Campus, Bagayam, Vellore, Tamil Nadu 632002, India., 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, Manipal, 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., Sukumaran D; Department of Integrative Biology, School of Bioscience and Technology, Vellore Institute of Technology (VIT, Deemed to be University), Vellore, Tamil Nadu 632014, India., Gopinathan S; Centre for Stem Cell Research (a Unit of inStem, Bengaluru), Christian Medical College Campus, Bagayam, Vellore, Tamil Nadu 632002, India., Pai AA; Department of Haematology, Christian Medical College & Hospital, Vellore, Tamil Nadu 632 004, India., Nakamura Y; Cell Engineering Division, RIKEN BioResource Center, 3-1-1 Koyadai, Tsukuba, Ibaraki 305-0074, Japan., Balasubramanian P; Department of Haematology, Christian Medical College & Hospital, Vellore, Tamil Nadu 632 004, India., Ramalingam R; Department of Integrative Biology, School of Bioscience and Technology, Vellore Institute of Technology (VIT, Deemed to be University), Vellore, Tamil Nadu 632014, 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, Tamil Nadu 632 004, India., Corn JE; Innovative Genomics Institute, University of California, Berkeley, Berkeley, CA 94704, USA; Institute of Molecular Health Sciences, Department of Biology, Zurich, Switzerland., Mackay JP; School of Life and Environmental Sciences, University of Sydney, Sydney, NSW 2006, Australia., 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, Tamil Nadu 632 004, India., Crossley M; School of Biotechnology and Biomolecular Sciences, University of New South Wales, Sydney, NSW, Australia., Mohankumar KM; Centre for Stem Cell Research (a Unit of inStem, Bengaluru), Christian Medical College Campus, Bagayam, Vellore, Tamil Nadu 632002, India. Electronic address: mohankumarkm@cmcvellore.ac.in.
Source: Molecular therapy : the journal of the American Society of Gene Therapy [Mol Ther] 2024 Mar 06; Vol. 32 (3), pp. 663-677. Date of Electronic Publication: 2024 Jan 24.
Publication Type: Journal Article; Research Support, Non-U.S. Gov't
Journal Info: Publisher: Cell Press Country of Publication: United States NLM ID: 100890581 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1525-0024 (Electronic) Linking ISSN: 15250016 NLM ISO Abbreviation: Mol Ther Subsets: MEDLINE
Database: MEDLINE Ultimate
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  Data: Base editing of key residues in the BCL11A-XL-specific zinc finger domains derepresses fetal globin expression.
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  Data: <searchLink fieldCode="AU" term="%22Rajendiran+V%22">Rajendiran V</searchLink>; 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.<br /><searchLink fieldCode="AU" term="%22Devaraju+N%22">Devaraju N</searchLink>; 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, Manipal, Karnataka 576104, India.<br /><searchLink fieldCode="AU" term="%22Haddad+M%22">Haddad M</searchLink>; School of Biotechnology and Biomolecular Sciences, University of New South Wales, Sydney, NSW, Australia.<br /><searchLink fieldCode="AU" term="%22Ravi+NS%22">Ravi NS</searchLink>; 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.<br /><searchLink fieldCode="AU" term="%22Panigrahi+L%22">Panigrahi L</searchLink>; 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, Manipal, Karnataka 576104, India.<br /><searchLink fieldCode="AU" term="%22Paul+J%22">Paul J</searchLink>; 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, Manipal, Karnataka 576104, India.<br /><searchLink fieldCode="AU" term="%22Gopalakrishnan+C%22">Gopalakrishnan C</searchLink>; Department of Integrative Biology, School of Bioscience and Technology, Vellore Institute of Technology (VIT, Deemed to be University), Vellore, Tamil Nadu 632014, India.<br /><searchLink fieldCode="AU" term="%22Wyman+S%22">Wyman S</searchLink>; Innovative Genomics Institute, University of California, Berkeley, Berkeley, CA 94704, USA.<br /><searchLink fieldCode="AU" term="%22Ariudainambi+K%22">Ariudainambi K</searchLink>; Department of Biochemistry, Auxilium College, Vellore, Tamil Nadu 632006, India.<br /><searchLink fieldCode="AU" term="%22Mahalingam+G%22">Mahalingam G</searchLink>; Centre for Stem Cell Research (a Unit of inStem, Bengaluru), Christian Medical College Campus, Bagayam, Vellore, Tamil Nadu 632002, India.<br /><searchLink fieldCode="AU" term="%22Periyasami+Y%22">Periyasami Y</searchLink>; Centre for Stem Cell Research (a Unit of inStem, Bengaluru), Christian Medical College Campus, Bagayam, Vellore, Tamil Nadu 632002, India.<br /><searchLink fieldCode="AU" term="%22Prasad+K%22">Prasad K</searchLink>; 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, Manipal, Karnataka 576104, India.<br /><searchLink fieldCode="AU" term="%22George+A%22">George A</searchLink>; 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.<br /><searchLink fieldCode="AU" term="%22Sukumaran+D%22">Sukumaran D</searchLink>; Department of Integrative Biology, School of Bioscience and Technology, Vellore Institute of Technology (VIT, Deemed to be University), Vellore, Tamil Nadu 632014, India.<br /><searchLink fieldCode="AU" term="%22Gopinathan+S%22">Gopinathan S</searchLink>; Centre for Stem Cell Research (a Unit of inStem, Bengaluru), Christian Medical College Campus, Bagayam, Vellore, Tamil Nadu 632002, India.<br /><searchLink fieldCode="AU" term="%22Pai+AA%22">Pai AA</searchLink>; Department of Haematology, Christian Medical College & Hospital, Vellore, Tamil Nadu 632 004, India.<br /><searchLink fieldCode="AU" term="%22Nakamura+Y%22">Nakamura Y</searchLink>; Cell Engineering Division, RIKEN BioResource Center, 3-1-1 Koyadai, Tsukuba, Ibaraki 305-0074, Japan.<br /><searchLink fieldCode="AU" term="%22Balasubramanian+P%22">Balasubramanian P</searchLink>; Department of Haematology, Christian Medical College & Hospital, Vellore, Tamil Nadu 632 004, India.<br /><searchLink fieldCode="AU" term="%22Ramalingam+R%22">Ramalingam R</searchLink>; Department of Integrative Biology, School of Bioscience and Technology, Vellore Institute of Technology (VIT, Deemed to be University), Vellore, Tamil Nadu 632014, India.<br /><searchLink fieldCode="AU" term="%22Thangavel+S%22">Thangavel S</searchLink>; Centre for Stem Cell Research (a Unit of inStem, Bengaluru), Christian Medical College Campus, Bagayam, Vellore, Tamil Nadu 632002, India.<br /><searchLink fieldCode="AU" term="%22Velayudhan+SR%22">Velayudhan SR</searchLink>; 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, Tamil Nadu 632 004, India.<br /><searchLink fieldCode="AU" term="%22Corn+JE%22">Corn JE</searchLink>; Innovative Genomics Institute, University of California, Berkeley, Berkeley, CA 94704, USA; Institute of Molecular Health Sciences, Department of Biology, Zurich, Switzerland.<br /><searchLink fieldCode="AU" term="%22Mackay+JP%22">Mackay JP</searchLink>; School of Life and Environmental Sciences, University of Sydney, Sydney, NSW 2006, Australia.<br /><searchLink fieldCode="AU" term="%22Marepally+S%22">Marepally S</searchLink>; Centre for Stem Cell Research (a Unit of inStem, Bengaluru), Christian Medical College Campus, Bagayam, Vellore, Tamil Nadu 632002, India.<br /><searchLink fieldCode="AU" term="%22Srivastava+A%22">Srivastava A</searchLink>; 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, Tamil Nadu 632 004, India.<br /><searchLink fieldCode="AU" term="%22Crossley+M%22">Crossley M</searchLink>; School of Biotechnology and Biomolecular Sciences, University of New South Wales, Sydney, NSW, Australia.<br /><searchLink fieldCode="AU" term="%22Mohankumar+KM%22">Mohankumar KM</searchLink>; Centre for Stem Cell Research (a Unit of inStem, Bengaluru), Christian Medical College Campus, Bagayam, Vellore, Tamil Nadu 632002, India. Electronic address: mohankumarkm@cmcvellore.ac.in.
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