Genomics

Dataset Information

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Homo sapiens


ABSTRACT: Genetic correction and analysis of induced pluripotent stem cells from a patient with gyrate atrophy

PROVIDER: PRJNA136291 | ENA |

REPOSITORIES: ENA

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Publications

Genetic correction and analysis of induced pluripotent stem cells from a patient with gyrate atrophy.

Howden Sara E SE   Gore Athurva A   Li Zhe Z   Fung Ho-Lim HL   Nisler Benjamin S BS   Nie Jeff J   Chen Goukai G   McIntosh Brian E BE   Gulbranson Daniel R DR   Diol Nicole R NR   Taapken Seth M SM   Vereide David T DT   Montgomery Karen Dyer KD   Zhang Kun K   Gamm David M DM   Thomson James A JA  

Proceedings of the National Academy of Sciences of the United States of America 20110404 16


Gene-corrected patient-specific induced pluripotent stem (iPS) cells offer a unique approach to gene therapy. Here, we begin to assess whether the mutational load acquired during gene correction of iPS cells is compatible with use in the treatment of genetic causes of retinal degenerative disease. We isolated iPS cells free of transgene sequences from a patient with gyrate atrophy caused by a point mutation in the gene encoding ornithine-δ-aminotransferase (OAT) and used homologous recombination  ...[more]

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