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CRISPR-Cas9-Mediated ELANE Mutation Correction in Hematopoietic Stem and Progenitor Cells to Treat Severe Congenital Neutropenia |
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| Authors: | Ngoc Tung Tran Robin Graf Annika Wulf-Goldenberg Maria Stecklum Gabriele Strauß Ralf Kühn Christine Kocks Klaus Rajewsky Van Trung Chu |
| Institution: | 1. Immune Regulation and Cancer, Max Delbrück Center for Molecular Medicine in the Helmholtz Association, 13125 Berlin, Germany;2. EPO GmbH, 13125 Berlin, Germany;3. Helios Klinikum, 13125 Berlin, Germany;4. iPS Cell Based Disease Modeling, Max Delbrück Center for Molecular Medicine in the Helmholtz Association, 13125 Berlin, Germany;5. Transgenics, Max Delbrück Center for Molecular Medicine in the Helmholtz Association, 13125 Berlin, Germany |
| Abstract: |  |
| Keywords: | CRISPR-Cas9 ribonucleoprotein RNP adeno-associated virus AAV non-homologous end joining NHEJ homology-directed repair HDR hematopoietic stem and progenitor cells HSPCs gene therapy neutrophil elastase NE severe congenital neutropenia |
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