TALEN-Mediated Gene Editing of HBG in Human Hematopoietic Stem Cells Leads to Therapeutic Fetal Hemoglobin Induction

Autor: Lux, Christopher T., Pattabhi, Sowmya, Berger, Mason, Nourigat, Cynthia, Flowers, David A., Negre, Olivier, Humbert, Olivier, Yang, Julia G., Lee, Calvin, Jacoby, Kyle, Bernstein, Irwin, Kiem, Hans-Peter, Scharenberg, Andrew, Rawlings, David J.

Publikováno v: In Molecular Therapy - Methods & Clinical Development 15 March 2019 12:175-183

TALEN-Mediated Gene Editing of HBG in Human Hematopoietic Stem Cells Leads to Therapeutic Fetal Hemoglobin Induction.

Autor: Lux CT; Center for Immunity and Immunotherapies and the Program for Cell and Gene Therapy, Seattle Children's Research Institute, Seattle, WA 98101, USA.; Department of Pediatrics, University of Washington, Seattle, WA 98195, USA., Pattabhi S; Center for Immunity and Immunotherapies and the Program for Cell and Gene Therapy, Seattle Children's Research Institute, Seattle, WA 98101, USA., Berger M; Center for Immunity and Immunotherapies and the Program for Cell and Gene Therapy, Seattle Children's Research Institute, Seattle, WA 98101, USA., Nourigat C; Stem Cell and Gene Therapy Program, Fred Hutchinson Cancer Research Center, Seattle, WA 98109, USA., Flowers DA; Stem Cell and Gene Therapy Program, Fred Hutchinson Cancer Research Center, Seattle, WA 98109, USA., Negre O; bluebird bio, Inc., Cambridge MA 02142, USA., Humbert O; Stem Cell and Gene Therapy Program, Fred Hutchinson Cancer Research Center, Seattle, WA 98109, USA., Yang JG; bluebird bio, Inc., Cambridge MA 02142, USA., Lee C; bluebird bio, Inc., Cambridge MA 02142, USA., Jacoby K; Center for Immunity and Immunotherapies and the Program for Cell and Gene Therapy, Seattle Children's Research Institute, Seattle, WA 98101, USA., Bernstein I; Stem Cell and Gene Therapy Program, Fred Hutchinson Cancer Research Center, Seattle, WA 98109, USA., Kiem HP; Stem Cell and Gene Therapy Program, Fred Hutchinson Cancer Research Center, Seattle, WA 98109, USA.; Department of Medicine, University of Washington, Seattle, WA 98195, USA.; Department of Pathology, University of Washington, Seattle, WA 98195, USA., Scharenberg A; Center for Immunity and Immunotherapies and the Program for Cell and Gene Therapy, Seattle Children's Research Institute, Seattle, WA 98101, USA.; Department of Pediatrics, University of Washington, Seattle, WA 98195, USA.; Department of Immunology, University of Washington, Seattle, WA 98195, USA., Rawlings DJ; Center for Immunity and Immunotherapies and the Program for Cell and Gene Therapy, Seattle Children's Research Institute, Seattle, WA 98101, USA.; Department of Pediatrics, University of Washington, Seattle, WA 98195, USA.; Department of Immunology, University of Washington, Seattle, WA 98195, USA.

Publikováno v: Molecular therapy. Methods & clinical development [Mol Ther Methods Clin Dev] 2018 Dec 31; Vol. 12, pp. 175-183. Date of Electronic Publication: 2018 Dec 31 (Print Publication: 2019).