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Editing the core region in HPFH deletions alters fetal and adult globin expression for treatment of β-hemoglobinopathies.

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Title: Editing the core region in HPFH deletions alters fetal and adult globin expression for treatment of β-hemoglobinopathies.
Authors: Venkatesan V; Centre for Stem Cell Research (CSCR), A Unit of InStem Bengaluru, Christian Medical College Campus, Vellore, Tamil Nadu 632002, India.; Manipal Academy of Higher Education, Manipal 576104, Karnataka, India., Christopher AC; Centre for Stem Cell Research (CSCR), A Unit of InStem Bengaluru, Christian Medical College Campus, Vellore, Tamil Nadu 632002, India., Rhiel M; Institute for Transfusion Medicine and Gene Therapy, Medical Center - University of Freiburg, 79106 Freiburg, Germany.; Center for Chronic Immunodeficiency, Medical Faculty, University of Freiburg, 79106 Freiburg, Germany., Azhagiri MKK; Centre for Stem Cell Research (CSCR), A Unit of InStem Bengaluru, Christian Medical College Campus, Vellore, Tamil Nadu 632002, India.; Manipal Academy of Higher Education, Manipal 576104, Karnataka, India., Babu P; Centre for Stem Cell Research (CSCR), A Unit of InStem Bengaluru, Christian Medical College Campus, Vellore, Tamil Nadu 632002, India.; Manipal Academy of Higher Education, Manipal 576104, Karnataka, India., Walavalkar K; National Centre for Biological Sciences, Tata Institute of Fundamental Research, Bangalore, Karnataka 560065, India., Saravanan B; National Centre for Biological Sciences, Tata Institute of Fundamental Research, Bangalore, Karnataka 560065, India., Andrieux G; Institute of Medical Bioinformatics and Systems Medicine, Faculty of Medicine & Medical Center - University of Freiburg, 79106 Freiburg, Germany.; German Cancer Consortium (DKTK), Partner Site Freiburg and German Cancer Research Center (DKFZ), 69120 Heidelberg, Germany., Rangaraj S; Centre for Stem Cell Research (CSCR), A Unit of InStem Bengaluru, Christian Medical College Campus, Vellore, Tamil Nadu 632002, India., Srinivasan S; Centre for Stem Cell Research (CSCR), A Unit of InStem Bengaluru, Christian Medical College Campus, Vellore, Tamil Nadu 632002, India., Karuppusamy KV; Centre for Stem Cell Research (CSCR), A Unit of InStem Bengaluru, Christian Medical College Campus, Vellore, Tamil Nadu 632002, India.; Manipal Academy of Higher Education, Manipal 576104, Karnataka, India., Jacob A; Centre for Stem Cell Research (CSCR), A Unit of InStem Bengaluru, Christian Medical College Campus, Vellore, Tamil Nadu 632002, India., Bagchi A; Centre for Stem Cell Research (CSCR), A Unit of InStem Bengaluru, Christian Medical College Campus, Vellore, Tamil Nadu 632002, India., Pai AA; Department of Hematology, Christian Medical College, Vellore, Tamil Nadu 632004, India., Nakamura Y; Cell Engineering Division, RIKEN BioResource Research Center, Ibaraki 3050074, Japan., Kurita R; Cell Engineering Division, RIKEN BioResource Research Center, Ibaraki 3050074, Japan., Balasubramanian P; Department of Hematology, Christian Medical College, Vellore, Tamil Nadu 632004, India., Pai R; Department of Pathology, Christian Medical College, Vellore, Tamil Nadu 632004, India., Marepally SK; Centre for Stem Cell Research (CSCR), A Unit of InStem Bengaluru, Christian Medical College Campus, Vellore, Tamil Nadu 632002, India., Mohankumar KM; Centre for Stem Cell Research (CSCR), A Unit of InStem Bengaluru, Christian Medical College Campus, Vellore, Tamil Nadu 632002, India., Velayudhan SR; Centre for Stem Cell Research (CSCR), A Unit of InStem Bengaluru, Christian Medical College Campus, Vellore, Tamil Nadu 632002, India.; Department of Hematology, Christian Medical College, Vellore, Tamil Nadu 632004, India., Boerries M; Institute of Medical Bioinformatics and Systems Medicine, Faculty of Medicine & Medical Center - University of Freiburg, 79106 Freiburg, Germany.; German Cancer Consortium (DKTK), Partner Site Freiburg and German Cancer Research Center (DKFZ), 69120 Heidelberg, Germany., Notani D; National Centre for Biological Sciences, Tata Institute of Fundamental Research, Bangalore, Karnataka 560065, India., Cathomen T; Institute for Transfusion Medicine and Gene Therapy, Medical Center - University of Freiburg, 79106 Freiburg, Germany.; Center for Chronic Immunodeficiency, Medical Faculty, University of Freiburg, 79106 Freiburg, Germany., Srivastava A; Centre for Stem Cell Research (CSCR), A Unit of InStem Bengaluru, Christian Medical College Campus, Vellore, Tamil Nadu 632002, India.; Department of Hematology, Christian Medical College, Vellore, Tamil Nadu 632004, India., Thangavel S; Centre for Stem Cell Research (CSCR), A Unit of InStem Bengaluru, Christian Medical College Campus, Vellore, Tamil Nadu 632002, India.
Source: Molecular therapy. Nucleic acids [Mol Ther Nucleic Acids] 2023 Apr 26; Vol. 32, pp. 671-688. Date of Electronic Publication: 2023 Apr 26 (Print Publication: 2023).
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.2023.04.024