In Vivo Genome Editing Restores Dystrophin Expression and Cardiac Function in Dystrophic Mice.

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Title: In Vivo Genome Editing Restores Dystrophin Expression and Cardiac Function in Dystrophic Mice.
Authors: El Refaey M; From the Department of Surgery, Davis Heart and Lung Research Institute, Biomedical Sciences Graduate Program, Biophysics Graduate Program (M.E.R., L.X., Y.G., T.M.A.A., J.M., R.H.) and Department of Physiology and Cell Biology, Davis Heart and Lung Research Institute (B.D.C., P.J.M., P.M.L.J.), The Ohio State University Wexner Medical Center, Columbus; Genomics Shared Resource, The Ohio State University Comprehensive Cancer Center, Columbus (S.C.W., D.E.S.); and Department of Cancer Biology and Genetics (K.A., D.E.S.) and Human Cancer Genetics Program, and Department of Biomedical Informatics (adjunct) (D.E.S.), The Ohio State University, Columbus., Xu L; From the Department of Surgery, Davis Heart and Lung Research Institute, Biomedical Sciences Graduate Program, Biophysics Graduate Program (M.E.R., L.X., Y.G., T.M.A.A., J.M., R.H.) and Department of Physiology and Cell Biology, Davis Heart and Lung Research Institute (B.D.C., P.J.M., P.M.L.J.), The Ohio State University Wexner Medical Center, Columbus; Genomics Shared Resource, The Ohio State University Comprehensive Cancer Center, Columbus (S.C.W., D.E.S.); and Department of Cancer Biology and Genetics (K.A., D.E.S.) and Human Cancer Genetics Program, and Department of Biomedical Informatics (adjunct) (D.E.S.), The Ohio State University, Columbus., Gao Y; From the Department of Surgery, Davis Heart and Lung Research Institute, Biomedical Sciences Graduate Program, Biophysics Graduate Program (M.E.R., L.X., Y.G., T.M.A.A., J.M., R.H.) and Department of Physiology and Cell Biology, Davis Heart and Lung Research Institute (B.D.C., P.J.M., P.M.L.J.), The Ohio State University Wexner Medical Center, Columbus; Genomics Shared Resource, The Ohio State University Comprehensive Cancer Center, Columbus (S.C.W., D.E.S.); and Department of Cancer Biology and Genetics (K.A., D.E.S.) and Human Cancer Genetics Program, and Department of Biomedical Informatics (adjunct) (D.E.S.), The Ohio State University, Columbus., Canan BD; From the Department of Surgery, Davis Heart and Lung Research Institute, Biomedical Sciences Graduate Program, Biophysics Graduate Program (M.E.R., L.X., Y.G., T.M.A.A., J.M., R.H.) and Department of Physiology and Cell Biology, Davis Heart and Lung Research Institute (B.D.C., P.J.M., P.M.L.J.), The Ohio State University Wexner Medical Center, Columbus; Genomics Shared Resource, The Ohio State University Comprehensive Cancer Center, Columbus (S.C.W., D.E.S.); and Department of Cancer Biology and Genetics (K.A., D.E.S.) and Human Cancer Genetics Program, and Department of Biomedical Informatics (adjunct) (D.E.S.), The Ohio State University, Columbus., Adesanya TMA; From the Department of Surgery, Davis Heart and Lung Research Institute, Biomedical Sciences Graduate Program, Biophysics Graduate Program (M.E.R., L.X., Y.G., T.M.A.A., J.M., R.H.) and Department of Physiology and Cell Biology, Davis Heart and Lung Research Institute (B.D.C., P.J.M., P.M.L.J.), The Ohio State University Wexner Medical Center, Columbus; Genomics Shared Resource, The Ohio State University Comprehensive Cancer Center, Columbus (S.C.W., D.E.S.); and Department of Cancer Biology and Genetics (K.A., D.E.S.) and Human Cancer Genetics Program, and Department of Biomedical Informatics (adjunct) (D.E.S.), The Ohio State University, Columbus., Warner SC; From the Department of Surgery, Davis Heart and Lung Research Institute, Biomedical Sciences Graduate Program, Biophysics Graduate Program (M.E.R., L.X., Y.G., T.M.A.A., J.M., R.H.) and Department of Physiology and Cell Biology, Davis Heart and Lung Research Institute (B.D.C., P.J.M., P.M.L.J.), The Ohio State University Wexner Medical Center, Columbus; Genomics Shared Resource, The Ohio State University Comprehensive Cancer Center, Columbus (S.C.W., D.E.S.); and Department of Cancer Biology and Genetics (K.A., D.E.S.) and Human Cancer Genetics Program, and Department of Biomedical Informatics (adjunct) (D.E.S.), The Ohio State University, Columbus., Akagi K; From the Department of Surgery, Davis Heart and Lung Research Institute, Biomedical Sciences Graduate Program, Biophysics Graduate Program (M.E.R., L.X., Y.G., T.M.A.A., J.M., R.H.) and Department of Physiology and Cell Biology, Davis Heart and Lung Research Institute (B.D.C., P.J.M., P.M.L.J.), The Ohio State University Wexner Medical Center, Columbus; Genomics Shared Resource, The Ohio State University Comprehensive Cancer Center, Columbus (S.C.W., D.E.S.); and Department of Cancer Biology and Genetics (K.A., D.E.S.) and Human Cancer Genetics Program, and Department of Biomedical Informatics (adjunct) (D.E.S.), The Ohio State University, Columbus., Symer DE; From the Department of Surgery, Davis Heart and Lung Research Institute, Biomedical Sciences Graduate Program, Biophysics Graduate Program (M.E.R., L.X., Y.G., T.M.A.A., J.M., R.H.) and Department of Physiology and Cell Biology, Davis Heart and Lung Research Institute (B.D.C., P.J.M., P.M.L.J.), The Ohio State University Wexner Medical Center, Columbus; Genomics Shared Resource, The Ohio State University Comprehensive Cancer Center, Columbus (S.C.W., D.E.S.); and Department of Cancer Biology and Genetics (K.A., D.E.S.) and Human Cancer Genetics Program, and Department of Biomedical Informatics (adjunct) (D.E.S.), The Ohio State University, Columbus., Mohler PJ; From the Department of Surgery, Davis Heart and Lung Research Institute, Biomedical Sciences Graduate Program, Biophysics Graduate Program (M.E.R., L.X., Y.G., T.M.A.A., J.M., R.H.) and Department of Physiology and Cell Biology, Davis Heart and Lung Research Institute (B.D.C., P.J.M., P.M.L.J.), The Ohio State University Wexner Medical Center, Columbus; Genomics Shared Resource, The Ohio State University Comprehensive Cancer Center, Columbus (S.C.W., D.E.S.); and Department of Cancer Biology and Genetics (K.A., D.E.S.) and Human Cancer Genetics Program, and Department of Biomedical Informatics (adjunct) (D.E.S.), The Ohio State University, Columbus., Ma J; From the Department of Surgery, Davis Heart and Lung Research Institute, Biomedical Sciences Graduate Program, Biophysics Graduate Program (M.E.R., L.X., Y.G., T.M.A.A., J.M., R.H.) and Department of Physiology and Cell Biology, Davis Heart and Lung Research Institute (B.D.C., P.J.M., P.M.L.J.), The Ohio State University Wexner Medical Center, Columbus; Genomics Shared Resource, The Ohio State University Comprehensive Cancer Center, Columbus (S.C.W., D.E.S.); and Department of Cancer Biology and Genetics (K.A., D.E.S.) and Human Cancer Genetics Program, and Department of Biomedical Informatics (adjunct) (D.E.S.), The Ohio State University, Columbus., Janssen PML; From the Department of Surgery, Davis Heart and Lung Research Institute, Biomedical Sciences Graduate Program, Biophysics Graduate Program (M.E.R., L.X., Y.G., T.M.A.A., J.M., R.H.) and Department of Physiology and Cell Biology, Davis Heart and Lung Research Institute (B.D.C., P.J.M., P.M.L.J.), The Ohio State University Wexner Medical Center, Columbus; Genomics Shared Resource, The Ohio State University Comprehensive Cancer Center, Columbus (S.C.W., D.E.S.); and Department of Cancer Biology and Genetics (K.A., D.E.S.) and Human Cancer Genetics Program, and Department of Biomedical Informatics (adjunct) (D.E.S.), The Ohio State University, Columbus., Han R; From the Department of Surgery, Davis Heart and Lung Research Institute, Biomedical Sciences Graduate Program, Biophysics Graduate Program (M.E.R., L.X., Y.G., T.M.A.A., J.M., R.H.) and Department of Physiology and Cell Biology, Davis Heart and Lung Research Institute (B.D.C., P.J.M., P.M.L.J.), The Ohio State University Wexner Medical Center, Columbus; Genomics Shared Resource, The Ohio State University Comprehensive Cancer Center, Columbus (S.C.W., D.E.S.); and Department of Cancer Biology and Genetics (K.A., D.E.S.) and Human Cancer Genetics Program, and Department of Biomedical Informatics (adjunct) (D.E.S.), The Ohio State University, Columbus. renzhi.han@osumc.edu.
Source: Circulation research [Circ Res] 2017 Sep 29; Vol. 121 (8), pp. 923-929. Date of Electronic Publication: 2017 Aug 08.
Publication Type: Journal Article
Journal Info: Publisher: Lippincott Williams & Wilkins Country of Publication: United States NLM ID: 0047103 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1524-4571 (Electronic) Linking ISSN: 00097330 NLM ISO Abbreviation: Circ Res Subsets: MEDLINE
Database: MEDLINE Ultimate
Description
ISSN:1524-4571
DOI:10.1161/CIRCRESAHA.117.310996