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CRISPR-Cas9-Mediated ELANE Mutation Correction in Hematopoietic Stem and Progenitor Cells to Treat Severe Congenital Neutropenia.
Tran, Ngoc Tung; Graf, Robin; Wulf-Goldenberg, Annika; Stecklum, Maria; Strauß, Gabriele; Kühn, Ralf; Kocks, Christine; Rajewsky, Klaus; Chu, Van Trung.
Affiliation
  • Tran NT; Immune Regulation and Cancer, Max Delbrück Center for Molecular Medicine in the Helmholtz Association, 13125 Berlin, Germany.
  • Graf R; Immune Regulation and Cancer, Max Delbrück Center for Molecular Medicine in the Helmholtz Association, 13125 Berlin, Germany.
  • Wulf-Goldenberg A; EPO GmbH, 13125 Berlin, Germany.
  • Stecklum M; EPO GmbH, 13125 Berlin, Germany.
  • Strauß G; Helios Klinikum, 13125 Berlin, Germany.
  • Kühn R; iPS Cell Based Disease Modeling, Max Delbrück Center for Molecular Medicine in the Helmholtz Association, 13125 Berlin, Germany.
  • Kocks C; Immune Regulation and Cancer, Max Delbrück Center for Molecular Medicine in the Helmholtz Association, 13125 Berlin, Germany; Transgenics, Max Delbrück Center for Molecular Medicine in the Helmholtz Association, 13125 Berlin, Germany.
  • Rajewsky K; Immune Regulation and Cancer, Max Delbrück Center for Molecular Medicine in the Helmholtz Association, 13125 Berlin, Germany. Electronic address: klaus.rajewsky@mdc-berlin.de.
  • Chu VT; Immune Regulation and Cancer, Max Delbrück Center for Molecular Medicine in the Helmholtz Association, 13125 Berlin, Germany; iPS Cell Based Disease Modeling, Max Delbrück Center for Molecular Medicine in the Helmholtz Association, 13125 Berlin, Germany. Electronic address: vantrung.chu@mdc-berlin.d
Mol Ther ; 28(12): 2621-2634, 2020 12 02.
Article in En | MEDLINE | ID: mdl-32822592
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Full text: 1 Database: MEDLINE Main subject: Genetic Therapy / Hematopoietic Stem Cell Transplantation / Leukocyte Elastase / CRISPR-Cas Systems / Congenital Bone Marrow Failure Syndromes / Mutation / Neutropenia Limits: Animals / Humans Language: En Journal: Mol Ther Journal subject: BIOLOGIA MOLECULAR / TERAPEUTICA Year: 2020 Type: Article Affiliation country: Germany
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Full text: 1 Database: MEDLINE Main subject: Genetic Therapy / Hematopoietic Stem Cell Transplantation / Leukocyte Elastase / CRISPR-Cas Systems / Congenital Bone Marrow Failure Syndromes / Mutation / Neutropenia Limits: Animals / Humans Language: En Journal: Mol Ther Journal subject: BIOLOGIA MOLECULAR / TERAPEUTICA Year: 2020 Type: Article Affiliation country: Germany