α-Globin mutations and Genetic Variants in γ-globin Promoters are Associated with Unelevated Hemoglobin F Expression of Atypical β ⁰ -thalassemia/HbE.

Autor: Satthakarn S; Faculty of Allied Health Sciences, Burapha University, Chonburi, Thailand., Panyasai S; Department of Medical Technology, School of Allied Health Sciences, University of Phayao, Phayao, Thailand. Electronic address: sitthichai.pa@up.ac.th.

Publikováno v: Archives of medical research [Arch Med Res] 2024 Sep; Vol. 55 (6), pp. 103055. Date of Electronic Publication: 2024 Jul 18.

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

Autor: 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.

Publikováno v: 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.

Effects of Mithramycin on BCL11A Gene Expression and on the Interaction of the BCL11A Transcriptional Complex to γ-Globin Gene Promoter Sequences.

Autor: Finotti A; Department of Life Sciences and Biotechnology, Section of Biochemistry and Molecular Biology, Ferrara University, 44121 Ferrara, Italy., Gasparello J; Department of Life Sciences and Biotechnology, Section of Biochemistry and Molecular Biology, Ferrara University, 44121 Ferrara, Italy., Zuccato C; Department of Life Sciences and Biotechnology, Section of Biochemistry and Molecular Biology, Ferrara University, 44121 Ferrara, Italy., Cosenza LC; Department of Life Sciences and Biotechnology, Section of Biochemistry and Molecular Biology, Ferrara University, 44121 Ferrara, Italy., Fabbri E; Department of Life Sciences and Biotechnology, Section of Biochemistry and Molecular Biology, Ferrara University, 44121 Ferrara, Italy., Bianchi N; Department of Life Sciences and Biotechnology, Section of Biochemistry and Molecular Biology, Ferrara University, 44121 Ferrara, Italy.; Department of Translational Medicine and for Romagna, Ferrara University, 44121 Ferrara, Italy., Gambari R; Center 'Chiara Gemmo and Elio Zago' for the Research on Thalassemia, Ferrara University, 44121 Ferrara, Italy.

Publikováno v: Genes [Genes (Basel)] 2023 Oct 11; Vol. 14 (10). Date of Electronic Publication: 2023 Oct 11.

Development and IND-enabling studies of a novel Cas9 genome-edited autologous CD34 ⁺ cell therapy to induce fetal hemoglobin for sickle cell disease.

Autor: Katta V; Department of Hematology, St. Jude Children's Research Hospital, Memphis, TN, USA., O'Keefe K; Department of Hematology, St. Jude Children's Research Hospital, Memphis, TN, USA., Li Y; Department of Hematology, St. Jude Children's Research Hospital, Memphis, TN, USA., Mayuranathan T; Department of Hematology, St. Jude Children's Research Hospital, Memphis, TN, USA., Lazzarotto CR; Department of Hematology, St. Jude Children's Research Hospital, Memphis, TN, USA., Wood RK; Department of Hematology, St. Jude Children's Research Hospital, Memphis, TN, USA., Levine RM; Department of Hematology, St. Jude Children's Research Hospital, Memphis, TN, USA., Powers A; Children's GMP LLC, St. Jude Children's Research Hospital, Memphis, TN, USA., Mayberry K; Department of Hematology, St. Jude Children's Research Hospital, Memphis, TN, USA., Manquen G; Department of Hematology, St. Jude Children's Research Hospital, Memphis, TN, USA., Yao Y; Department of Hematology, St. Jude Children's Research Hospital, Memphis, TN, USA., Zhang J; Department of Hematology, St. Jude Children's Research Hospital, Memphis, TN, USA., Jang Y; Department of Hematology, St. Jude Children's Research Hospital, Memphis, TN, USA., Nimmagadda N; Department of Hematology, St. Jude Children's Research Hospital, Memphis, TN, USA., Dempsey EA; Department of Hematology, St. Jude Children's Research Hospital, Memphis, TN, USA., Lee G; Department of Hematology, St. Jude Children's Research Hospital, Memphis, TN, USA., Uchida N; Cellular and Molecular Therapeutics Branch, National Heart, Lung, and Blood Institute/National Institute of Diabetes and Digestive and Kidney Diseases, National Institute of Health Bethesda, Bethesda, MD, USA., Cheng Y; Department of Hematology, St. Jude Children's Research Hospital, Memphis, TN, USA., Fazio F; Children's GMP LLC, St. Jude Children's Research Hospital, Memphis, TN, USA., Lockey T; Children's GMP LLC, St. Jude Children's Research Hospital, Memphis, TN, USA., Meagher M; Children's GMP LLC, St. Jude Children's Research Hospital, Memphis, TN, USA., Sharma A; Department of Bone Marrow Transplantation & Cellular Therapy, St. Jude Children's Research Hospital, Memphis, TN, USA., Tisdale JF; Cellular and Molecular Therapeutics Branch, National Heart, Lung, and Blood Institute/National Institute of Diabetes and Digestive and Kidney Diseases, National Institute of Health Bethesda, Bethesda, MD, USA., Zhou S; Experimental & Cellular Therapeutics Lab, St. Jude Children's Research Hospital, Memphis, TN, USA., Yen JS; Department of Hematology, St. Jude Children's Research Hospital, Memphis, TN, USA. Electronic address: jonathan.yen@stjude.org., Weiss MJ; Department of Hematology, St. Jude Children's Research Hospital, Memphis, TN, USA. Electronic address: mitch.weiss@stjude.org., Tsai SQ; Department of Hematology, St. Jude Children's Research Hospital, Memphis, TN, USA. Electronic address: shengdar.tsai@stjude.org.

Publikováno v: Molecular therapy : the journal of the American Society of Gene Therapy [Mol Ther] 2024 Oct 02; Vol. 32 (10), pp. 3433-3452. Date of Electronic Publication: 2024 Jul 31.

Combinatorial targeting of epigenome-modifying enzymes with decitabine and RN-1 synergistically increases HbF.

Autor: Ibanez V; Section of Hematology/Oncology, Department of Medicine, University of Illinois at Chicago, Chicago, IL.; Research and Development, Jesse Brown VA Medical Center, Chicago, IL., Vaitkus K; Section of Hematology/Oncology, Department of Medicine, University of Illinois at Chicago, Chicago, IL.; Research and Development, Jesse Brown VA Medical Center, Chicago, IL., Zhang X; Section of Hematology/Oncology, Department of Medicine, University of Illinois at Chicago, Chicago, IL., Ramasamy J; Department of Pediatrics, University of Illinois at Chicago, Chicago, IL., Rivers AE; Department of Pediatrics, School of Medicine, University of California at San Francisco Benioff Children's Hospital Oakland, Oakland, CA., Saunthararajah Y; Department of Hematologic Oncology and Blood Disorders, Taussig Cancer Center, Cleveland Clinic, Cleveland, OH., Molokie R; Section of Hematology/Oncology, Department of Medicine, University of Illinois at Chicago, Chicago, IL.; Research and Development, Jesse Brown VA Medical Center, Chicago, IL.; Department of Pharmaceutical Science, University of Illinois at Chicago, Chicago, IL., Lavelle D; Section of Hematology/Oncology, Department of Medicine, University of Illinois at Chicago, Chicago, IL.; Research and Development, Jesse Brown VA Medical Center, Chicago, IL.

Publikováno v: Blood advances [Blood Adv] 2023 Aug 08; Vol. 7 (15), pp. 3891-3902.