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Evaluation of CRISPR/Cas9 exon-skipping vector for choroideremia using human induced pluripotent stem cell-derived RPE |
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| Authors: | Toshiro Iwagawa Hiroki Masumoto Hitoshi Tabuchi Kenzaburo Tani Bruce R Conklin Sumiko Watanabe |
| Institution: | 1. Division of Molecular and Developmental Biology, Institute of Medical Science, University of Tokyo, Tokyo, Japan
Department of Retinal Biology and Pathology, University of Tokyo Hospital, University of Tokyo, Tokyo, Japan;2. Xeno Hoc, Inc., Tokyo, Japan Department of Ophthalmology, Tsukazaki Hospital, Himeji, Hyogo, Japan;3. Department of Ophthalmology, Tsukazaki Hospital, Himeji, Hyogo, Japan Department of Technology and Design Thinking for Medicine, Hiroshima University, Hiroshima, Japan;4. Laboratory of ALA Advanced Medical Research, Institute for quantitative Biosciences, University of Tokyo, Tokyo, Japan;5. Gladstone Institutes, San Francisco, CA, USA Departments of Medicine, Ophthalmology and Cellular and Molecular Pharmacology University of California, San Francisco, CA, USA;6. Division of Molecular and Developmental Biology, Institute of Medical Science, University of Tokyo, Tokyo, Japan |
| Abstract: | |
| Keywords: | CHM choroideremia CRISPR/Cas9 exon-skipping hiPSC RPE |