The Combination of CRISPR/Cas9 and iPSC Technologies in the Gene Therapy of Human β-thalassemia in Mice. [electronic resource]
Producer: 20180516Description: 32463 p. digitalISSN:- 2045-2322
- Animals
- CRISPR-Cas Systems
- Cell Differentiation
- Female
- Gene Editing -- methods
- Gene Expression
- Genetic Therapy -- methods
- Hematopoietic Stem Cell Transplantation
- Hematopoietic Stem Cells -- cytology
- Homologous Recombination
- Humans
- Induced Pluripotent Stem Cells -- metabolism
- Interleukin Receptor Common gamma Subunit -- deficiency
- Mice
- Mice, Inbred NOD
- Mice, SCID
- Mutation
- Whole-Body Irradiation
- beta-Globins -- deficiency
- beta-Thalassemia -- genetics
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Publication Type: Journal Article; Research Support, Non-U.S. Gov't
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