CRISPR/Cas9 β-globin gene targeting in human haematopoietic stem cells.

Dever, Daniel P

CRISPR/Cas9 β-globin gene targeting in human haematopoietic stem cells. [electronic resource] - Nature 11 2016 - 384-389 p. digital

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

1476-4687

10.1038/nature20134 doi


Alleles
Anemia, Sickle Cell--genetics
Animals
Antigens, CD34--metabolism
CRISPR-Associated Proteins--metabolism
CRISPR-Cas Systems--genetics
Cell Differentiation
Cell Lineage
Cell Separation
Dependovirus--genetics
Erythrocytes
Female
Flow Cytometry
Gene Editing--methods
Gene Targeting
Genes, Reporter
Genetic Therapy--methods
Hematopoietic Stem Cells--metabolism
Homologous Recombination
Humans
Magnets
Mice, Inbred NOD
Mice, SCID
Microspheres
Mutation
RNA, Messenger--biosynthesis
beta-Globins--genetics
beta-Thalassemia--genetics