The Combination of CRISPR/Cas9 and iPSC Technologies in the Gene Therapy of Human β-thalassemia in Mice.

Ou, Zhanhui

The Combination of CRISPR/Cas9 and iPSC Technologies in the Gene Therapy of Human β-thalassemia in Mice. [electronic resource] - Scientific reports 09 2016 - 32463 p. digital

Publication Type: Journal Article; Research Support, Non-U.S. Gov't

2045-2322

10.1038/srep32463 doi


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