RONALD RAINGER (1949–2016)

Autor: Bengtsson (né Benson), Keith R.

Publikováno v: Isis, 2017 Sep 01. 108(3), 654-656.

Externí odkaz: https://www.jstor.org/stable/26456321

Comparison of dystrophin expression following gene editing and gene replacement in an aged preclinical DMD animal model.

Autor: Bengtsson NE; Department of Neurology, University of Washington School of Medicine, Seattle, WA 98109-8055, USA; Senator Paul D. Wellstone Muscular Dystrophy Specialized Research Center, University of Washington School of Medicine, Seattle, WA 98109-8055, USA. Electronic address: niclasb@uw.edu., Crudele JM; Department of Neurology, University of Washington School of Medicine, Seattle, WA 98109-8055, USA; Senator Paul D. Wellstone Muscular Dystrophy Specialized Research Center, University of Washington School of Medicine, Seattle, WA 98109-8055, USA., Klaiman JM; Senator Paul D. Wellstone Muscular Dystrophy Specialized Research Center, University of Washington School of Medicine, Seattle, WA 98109-8055, USA; Department of Rehabilitation Medicine, University of Washington School of Medicine, Seattle, WA 98109-8055, USA., Halbert CL; Department of Neurology, University of Washington School of Medicine, Seattle, WA 98109-8055, USA; Senator Paul D. Wellstone Muscular Dystrophy Specialized Research Center, University of Washington School of Medicine, Seattle, WA 98109-8055, USA., Hauschka SD; Senator Paul D. Wellstone Muscular Dystrophy Specialized Research Center, University of Washington School of Medicine, Seattle, WA 98109-8055, USA; Department of Biochemistry, University of Washington School of Medicine, Seattle, WA 98109-8055, USA., Chamberlain JS; Department of Neurology, University of Washington School of Medicine, Seattle, WA 98109-8055, USA; Senator Paul D. Wellstone Muscular Dystrophy Specialized Research Center, University of Washington School of Medicine, Seattle, WA 98109-8055, USA; Department of Biochemistry, University of Washington School of Medicine, Seattle, WA 98109-8055, USA; Department of Medicine, University of Washington School of Medicine, Seattle, WA 98109-8055, USA.

Publikováno v: Molecular therapy : the journal of the American Society of Gene Therapy [Mol Ther] 2022 Jun 01; Vol. 30 (6), pp. 2176-2185. Date of Electronic Publication: 2022 Feb 08.

High levels of AAV vector integration into CRISPR-induced DNA breaks.

Autor: Hanlon KS; Department of Neurobiology, Harvard Medical School, Boston, MA, 02115, USA.; Molecular Neurogenetics Unit, Department of Neurology, Massachusetts General Hospital, Charlestown, MA, 02129, USA., Kleinstiver BP; Center for Genomic Medicine, Massachusetts General Hospital, Boston, MA, USA.; Department of Pathology, Massachusetts General Hospital, Boston, MA, USA.; Department of Pathology, Harvard Medical School, Boston, MA, USA., Garcia SP; Department of Pathology, Harvard Medical School, Boston, MA, USA.; Molecular Pathology Unit, Massachusetts General Hospital, Charlestown, MA, 02129, USA.; Center for Cancer Research and Center for Computational and Integrative Biology, Massachusetts General Hospital, Charlestown, MA, USA., Zaborowski MP; Molecular Neurogenetics Unit, Department of Neurology, Massachusetts General Hospital, Charlestown, MA, 02129, USA.; Program in Neuroscience, Harvard Medical School, Boston, MA, 02115, USA.; Department of Gynecology, Obstetrics and Gynecologic Oncology, Division of Gynecologic Oncology, Poznań University of Medical Sciences, 60-535, Poznań, Poland., Volak A; Molecular Neurogenetics Unit, Department of Neurology, Massachusetts General Hospital, Charlestown, MA, 02129, USA.; Institute of Molecular and Clinical Ophthalmology Basel, 4031, Basel, Switzerland., Spirig SE; Institute of Molecular and Clinical Ophthalmology Basel, 4031, Basel, Switzerland., Muller A; Institute of Molecular and Clinical Ophthalmology Basel, 4031, Basel, Switzerland., Sousa AA; Molecular Pathology Unit, Massachusetts General Hospital, Charlestown, MA, 02129, USA.; Center for Cancer Research and Center for Computational and Integrative Biology, Massachusetts General Hospital, Charlestown, MA, USA., Tsai SQ; Department of Hematology, St. Jude Children's Research Hospital, Memphis, TN, USA., Bengtsson NE; Department of Neurology, University of Washington, Seattle, WA, 98195, USA., Lööv C; Uppsala University, Department of Public Health and Caring Sciences, Geriatrics, Uppsala, Sweden., Ingelsson M; Uppsala University, Department of Public Health and Caring Sciences, Geriatrics, Uppsala, Sweden., Chamberlain JS; Department of Neurology, University of Washington, Seattle, WA, 98195, USA., Corey DP; Department of Neurobiology, Harvard Medical School, Boston, MA, 02115, USA., Aryee MJ; Department of Pathology, Harvard Medical School, Boston, MA, USA.; Molecular Pathology Unit, Massachusetts General Hospital, Charlestown, MA, 02129, USA.; Center for Cancer Research and Center for Computational and Integrative Biology, Massachusetts General Hospital, Charlestown, MA, USA.; Department of Biostatistics, Harvard T. H. Chan School of Public Health, Boston, MA, USA., Joung JK; Department of Pathology, Harvard Medical School, Boston, MA, USA.; Molecular Pathology Unit, Massachusetts General Hospital, Charlestown, MA, 02129, USA.; Center for Cancer Research and Center for Computational and Integrative Biology, Massachusetts General Hospital, Charlestown, MA, USA., Breakefield XO; Molecular Neurogenetics Unit, Department of Neurology, Massachusetts General Hospital, Charlestown, MA, 02129, USA.; Program in Neuroscience, Harvard Medical School, Boston, MA, 02115, USA., Maguire CA; Molecular Neurogenetics Unit, Department of Neurology, Massachusetts General Hospital, Charlestown, MA, 02129, USA. cmaguire@mgh.harvard.edu.; Program in Neuroscience, Harvard Medical School, Boston, MA, 02115, USA. cmaguire@mgh.harvard.edu., György B; Department of Neurobiology, Harvard Medical School, Boston, MA, 02115, USA. bence.gyoergy@iob.ch.; Molecular Neurogenetics Unit, Department of Neurology, Massachusetts General Hospital, Charlestown, MA, 02129, USA. bence.gyoergy@iob.ch.; Institute of Molecular and Clinical Ophthalmology Basel, 4031, Basel, Switzerland. bence.gyoergy@iob.ch.

Publikováno v: Nature communications [Nat Commun] 2019 Sep 30; Vol. 10 (1), pp. 4439. Date of Electronic Publication: 2019 Sep 30.

Non-invasive tracking of disease progression in young dystrophic muscles using multi-parametric MRI at 14T.

Autor: Park JS; Department of Radiology, University of Washington, Seattle, WA, United States of America., Vohra R; Department of Radiology, University of Washington, Seattle, WA, United States of America., Klussmann T; Department of Radiology, University of Washington, Seattle, WA, United States of America., Bengtsson NE; Department of Neurology, University of Washington, Seattle, WA, United States of America.; Senator Paul D. Wellstone Muscular Dystrophy Cooperative Research Center, University of Washington, Seattle, WA, United States of America., Chamberlain JS; Department of Neurology, University of Washington, Seattle, WA, United States of America.; Senator Paul D. Wellstone Muscular Dystrophy Cooperative Research Center, University of Washington, Seattle, WA, United States of America.; Department of Biochemistry, University of Washington, Seattle, WA, United States of America.; Department of Medicine, University of Washington, Seattle, WA, United States of America., Lee D; Department of Radiology, University of Washington, Seattle, WA, United States of America.

Publikováno v: PloS one [PLoS One] 2018 Oct 26; Vol. 13 (10), pp. e0206323. Date of Electronic Publication: 2018 Oct 26 (Print Publication: 2018).

Muscle-specific CRISPR/Cas9 dystrophin gene editing ameliorates pathophysiology in a mouse model for Duchenne muscular dystrophy.

Autor: Bengtsson NE; Department of Neurology, University of Washington, Seattle, Washington 98195-7720, USA.; Senator Paul D. Wellstone Muscular Dystrophy Cooperative Research Center, University of Washington, Seattle, Washington 98195-7720, USA., Hall JK; Department of Neurology, University of Washington, Seattle, Washington 98195-7720, USA.; Senator Paul D. Wellstone Muscular Dystrophy Cooperative Research Center, University of Washington, Seattle, Washington 98195-7720, USA., Odom GL; Department of Neurology, University of Washington, Seattle, Washington 98195-7720, USA.; Senator Paul D. Wellstone Muscular Dystrophy Cooperative Research Center, University of Washington, Seattle, Washington 98195-7720, USA., Phelps MP; Department of Pathology, University of Washington, Seattle, Washington 98195-7720, USA., Andrus CR; Department of Medicine, University of Washington, Seattle, Washington 98195-7720, USA.; Department of Genome Sciences, University of Washington, Seattle, Washington 98195-7720, USA., Hawkins RD; Department of Medicine, University of Washington, Seattle, Washington 98195-7720, USA.; Department of Genome Sciences, University of Washington, Seattle, Washington 98195-7720, USA., Hauschka SD; Senator Paul D. Wellstone Muscular Dystrophy Cooperative Research Center, University of Washington, Seattle, Washington 98195-7720, USA.; Department of Biochemistry, University of Washington, Seattle, Washington 98195-7720, USA., Chamberlain JR; Senator Paul D. Wellstone Muscular Dystrophy Cooperative Research Center, University of Washington, Seattle, Washington 98195-7720, USA.; Department of Medicine, University of Washington, Seattle, Washington 98195-7720, USA., Chamberlain JS; Department of Neurology, University of Washington, Seattle, Washington 98195-7720, USA.; Senator Paul D. Wellstone Muscular Dystrophy Cooperative Research Center, University of Washington, Seattle, Washington 98195-7720, USA.; Department of Medicine, University of Washington, Seattle, Washington 98195-7720, USA.; Department of Biochemistry, University of Washington, Seattle, Washington 98195-7720, USA.

Publikováno v: Nature communications [Nat Commun] 2017 Feb 14; Vol. 8, pp. 14454. Date of Electronic Publication: 2017 Feb 14.