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This vector has potential for targeted gene correction in hemoglobinopathies.
Yusa, K. et al. Targeted gene correction of α1-antitrypsin deficiency in induced pluripotent stem cells.
Highly efficient endogenous human gene correction using designed zinc-finger nucleases.
Urnov, F. D. et al. Highly efficient endogenous human gene correction using designed zinc-finger nucleases.
Aartsma-Rus, A. et al. Targeted exon skipping as a potential gene correction therapy for Duchenne muscular dystrophy.
Davis, L. & Maizels, N. Two distinct pathways support gene correction by single-stranded donors at DNA nicks.
He thinks that the issue will be settled only when others attempt gene correction in human embryos.
Lachmann, N. et al. Gene correction of human induced pluripotent stem cells repairs the cellular phenotype in pulmonary alveolar proteinosis.
In this work, repair-PPRHs are shown to be a new powerful tool for gene correction.
This communication discusses the possibility of applying targeted gene correction to dystrophic muscle in Duchenne dystrophy.
These findings were phenocopied in gene-edited homozygous SHANK2 knockout cells and rescued by gene correction of an ASD SHANK2 mutation.
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