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1Division of Hematology, Department of Internal Medicine,Medical University of Graz, 2Division of Hematology, Stanford Cancer Institute, Stanford Institute for Stem Cell Biology and Regenerative Medicine,Stanford University School of Medicine, 3Department of Blood Group Serology and Transfusion Medicine,Medical University of Graz
Chapters
- 00:05Introduction
- 00:54sgRNA Design
- 01:57Homology-Directed Repair Vector Construction
- 05:42Editing of Hematopoietic Stem and Progenitor Cells
- 09:48Results: Heterozygous Gain-of-Function Mutations in Hematopoietic Stem and Progenitor Cells by Combining the Use of CRISPR/Cas9 and Dual rAAV Donor Transduction
- 11:19Conclusion
