Zinc-finger nuclease-mediated correction of α-thalassemia in iPS cells.
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ABSTRACT:
SUBMITTER: Chang CJ
PROVIDER: S-EPMC3543982 | biostudies-literature | 2012 Nov
REPOSITORIES: biostudies-literature
Publications
Zinc-finger nuclease-mediated correction of α-thalassemia in iPS cells.
Chang Chan-Jung CJ Bouhassira Eric E EE
Blood 20120921 19
Induced pluripotent stem (iPS) cell technology holds vast promises for a cure to the hemoglobinopathies. Constructs and methods to safely insert therapeutic genes to correct the genetic defect need to be developed. Site-specific insertion is a very attractive method for gene therapy because the risks of insertional mutagenesis are eliminated provided that a "safe harbor" is identified, and because a single set of validated constructs can be used to correct a large variety of mutations simplifyin ...[more]
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