Ontology highlight
ABSTRACT:
SUBMITTER: Voit RA
PROVIDER: S-EPMC3902937 | biostudies-literature | 2014 Jan
REPOSITORIES: biostudies-literature
Voit Richard A RA Hendel Ayal A Pruett-Miller Shondra M SM Porteus Matthew H MH
Nucleic acids research 20131023 2
Tal-effector nucleases (TALENs) are engineered proteins that can stimulate precise genome editing through specific DNA double-strand breaks. Sickle cell disease and β-thalassemia are common genetic disorders caused by mutations in β-globin, and we engineered a pair of highly active TALENs that induce modification of 54% of human β-globin alleles near the site of the sickle mutation. These TALENS stimulate targeted integration of therapeutic, full-length beta-globin cDNA to the endogenous β-globi ...[more]