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Single-cut genome editing restores dystrophin expression in a new mouse model of muscular dystrophy.
Amoasii, Leonela; Long, Chengzu; Li, Hui; Mireault, Alex A; Shelton, John M; Sanchez-Ortiz, Efrain; McAnally, John R; Bhattacharyya, Samadrita; Schmidt, Florian; Grimm, Dirk; Hauschka, Stephen D; Bassel-Duby, Rhonda; Olson, Eric N.
Affiliation
  • Amoasii L; Department of Molecular Biology, Hamon Center for Regenerative Science and Medicine, Senator Paul D. Wellstone Muscular Dystrophy Cooperative Research Center, University of Texas Southern Medical Center, 5323 Harry Hines Boulevard, Dallas, TX 75390, USA.
  • Long C; Department of Molecular Biology, Hamon Center for Regenerative Science and Medicine, Senator Paul D. Wellstone Muscular Dystrophy Cooperative Research Center, University of Texas Southern Medical Center, 5323 Harry Hines Boulevard, Dallas, TX 75390, USA.
  • Li H; Department of Molecular Biology, Hamon Center for Regenerative Science and Medicine, Senator Paul D. Wellstone Muscular Dystrophy Cooperative Research Center, University of Texas Southern Medical Center, 5323 Harry Hines Boulevard, Dallas, TX 75390, USA.
  • Mireault AA; Department of Molecular Biology, Hamon Center for Regenerative Science and Medicine, Senator Paul D. Wellstone Muscular Dystrophy Cooperative Research Center, University of Texas Southern Medical Center, 5323 Harry Hines Boulevard, Dallas, TX 75390, USA.
  • Shelton JM; Department of Internal Medicine, University of Texas Southwestern Medical Center, Dallas, TX 75390, USA.
  • Sanchez-Ortiz E; Department of Molecular Biology, Hamon Center for Regenerative Science and Medicine, Senator Paul D. Wellstone Muscular Dystrophy Cooperative Research Center, University of Texas Southern Medical Center, 5323 Harry Hines Boulevard, Dallas, TX 75390, USA.
  • McAnally JR; Department of Molecular Biology, Hamon Center for Regenerative Science and Medicine, Senator Paul D. Wellstone Muscular Dystrophy Cooperative Research Center, University of Texas Southern Medical Center, 5323 Harry Hines Boulevard, Dallas, TX 75390, USA.
  • Bhattacharyya S; Department of Molecular Biology, Hamon Center for Regenerative Science and Medicine, Senator Paul D. Wellstone Muscular Dystrophy Cooperative Research Center, University of Texas Southern Medical Center, 5323 Harry Hines Boulevard, Dallas, TX 75390, USA.
  • Schmidt F; Heidelberg University Hospital, Center for Infectious Diseases, Virology, Cluster of Excellence Cell Networks, DZIF partner, BioQuant Center, Heidelberg D-69120, Germany.
  • Grimm D; Heidelberg University Hospital, Center for Infectious Diseases, Virology, Cluster of Excellence Cell Networks, DZIF partner, BioQuant Center, Heidelberg D-69120, Germany.
  • Hauschka SD; Department of Biochemistry, University of Washington, Seattle, WA 98195, USA.
  • Bassel-Duby R; Department of Molecular Biology, Hamon Center for Regenerative Science and Medicine, Senator Paul D. Wellstone Muscular Dystrophy Cooperative Research Center, University of Texas Southern Medical Center, 5323 Harry Hines Boulevard, Dallas, TX 75390, USA.
  • Olson EN; Department of Molecular Biology, Hamon Center for Regenerative Science and Medicine, Senator Paul D. Wellstone Muscular Dystrophy Cooperative Research Center, University of Texas Southern Medical Center, 5323 Harry Hines Boulevard, Dallas, TX 75390, USA. eric.olson@utsouthwestern.edu.
Sci Transl Med ; 9(418)2017 Nov 29.
Article in En | MEDLINE | ID: mdl-29187645
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Full text: 1 Collection: 01-internacional Database: MEDLINE Main subject: Dystrophin / Gene Editing Limits: Animals Language: En Journal: Sci Transl Med Year: 2017 Document type: Article
Fulltext
Print
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PubMed Links
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Full text: 1 Collection: 01-internacional Database: MEDLINE Main subject: Dystrophin / Gene Editing Limits: Animals Language: En Journal: Sci Transl Med Year: 2017 Document type: Article