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Akademický článek

A Single CRISPR-Cas9 Deletion Strategy that Targets the Majority of DMD Patients Restores Dystrophin Function in hiPSC-Derived Muscle Cells.

Autor: Young CS; Molecular Biology Institute, University of California, Los Angeles, CA 90095, USA; Center for Duchenne Muscular Dystrophy, University of California, Los Angeles, CA 90095, USA; Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research, University of California, Los Angeles, CA 90095, USA; Department of Neurology, University of California, Los Angeles, CA 90095, USA., Hicks MR; Center for Duchenne Muscular Dystrophy, University of California, Los Angeles, CA 90095, USA; Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research, University of California, Los Angeles, CA 90095, USA; Department of Microbiology, Immunology, and Molecular Genetics, University of California, Los Angeles, CA 90095, USA., Ermolova NV; Center for Duchenne Muscular Dystrophy, University of California, Los Angeles, CA 90095, USA; Department of Neurology, University of California, Los Angeles, CA 90095, USA., Nakano H; Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research, University of California, Los Angeles, CA 90095, USA; Department of Molecular, Cell, and Developmental Biology, University of California, Los Angeles, CA 90095, USA., Jan M; Center for Duchenne Muscular Dystrophy, University of California, Los Angeles, CA 90095, USA; Department of Microbiology, Immunology, and Molecular Genetics, University of California, Los Angeles, CA 90095, USA; CIRM Bridges Program, California State University, Northridge, CA 91330, USA., Younesi S; Center for Duchenne Muscular Dystrophy, University of California, Los Angeles, CA 90095, USA; Department of Microbiology, Immunology, and Molecular Genetics, University of California, Los Angeles, CA 90095, USA; CIRM Bridges Program, California State University, Northridge, CA 91330, USA., Karumbayaram S; Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research, University of California, Los Angeles, CA 90095, USA; Department of Microbiology, Immunology, and Molecular Genetics, University of California, Los Angeles, CA 90095, USA., Kumagai-Cresse C; Center for Duchenne Muscular Dystrophy, University of California, Los Angeles, CA 90095, USA; Department of Neurology, University of California, Los Angeles, CA 90095, USA., Wang D; Center for Duchenne Muscular Dystrophy, University of California, Los Angeles, CA 90095, USA; Department of Microbiology, Immunology, and Molecular Genetics, University of California, Los Angeles, CA 90095, USA., Zack JA; Molecular Biology Institute, University of California, Los Angeles, CA 90095, USA; Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research, University of California, Los Angeles, CA 90095, USA; Department of Microbiology, Immunology, and Molecular Genetics, University of California, Los Angeles, CA 90095, USA., Kohn DB; Molecular Biology Institute, University of California, Los Angeles, CA 90095, USA; Center for Duchenne Muscular Dystrophy, University of California, Los Angeles, CA 90095, USA; Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research, University of California, Los Angeles, CA 90095, USA; Department of Microbiology, Immunology, and Molecular Genetics, University of California, Los Angeles, CA 90095, USA., Nakano A; Molecular Biology Institute, University of California, Los Angeles, CA 90095, USA; Center for Duchenne Muscular Dystrophy, University of California, Los Angeles, CA 90095, USA; Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research, University of California, Los Angeles, CA 90095, USA; Department of Molecular, Cell, and Developmental Biology, University of California, Los Angeles, CA 90095, USA., Nelson SF; Molecular Biology Institute, University of California, Los Angeles, CA 90095, USA; Center for Duchenne Muscular Dystrophy, University of California, Los Angeles, CA 90095, USA; Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research, University of California, Los Angeles, CA 90095, USA; Department of Human Genetics, University of California, Los Angeles, CA 90095, USA., Miceli MC; Molecular Biology Institute, University of California, Los Angeles, CA 90095, USA; Center for Duchenne Muscular Dystrophy, University of California, Los Angeles, CA 90095, USA; Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research, University of California, Los Angeles, CA 90095, USA; Department of Microbiology, Immunology, and Molecular Genetics, University of California, Los Angeles, CA 90095, USA., Spencer MJ; Molecular Biology Institute, University of California, Los Angeles, CA 90095, USA; Center for Duchenne Muscular Dystrophy, University of California, Los Angeles, CA 90095, USA; Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research, University of California, Los Angeles, CA 90095, USA; Department of Neurology, University of California, Los Angeles, CA 90095, USA. Electronic address: mspencer@mednet.ucla.edu., Pyle AD; Molecular Biology Institute, University of California, Los Angeles, CA 90095, USA; Center for Duchenne Muscular Dystrophy, University of California, Los Angeles, CA 90095, USA; Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research, University of California, Los Angeles, CA 90095, USA; Department of Microbiology, Immunology, and Molecular Genetics, University of California, Los Angeles, CA 90095, USA. Electronic address: apyle@mednet.ucla.edu.

Publikováno v: Cell stem cell [Cell Stem Cell] 2016 Apr 07; Vol. 18 (4), pp. 533-40. Date of Electronic Publication: 2016 Feb 11.

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Akademický článek

A reporter mouse for optical imaging of inflammation in mdx muscles.

Autor: Martinez L; Department of Neurology and Center for Duchenne Muscular Dystrophy, David Geffen School of Medicine, University of California Los Angeles, 635 Charles Young Dr. South, NRB Room 401, Los Angeles, CA 90095 USA ; Center for Duchenne Muscular Dystrophy at UCLA, Los Angeles, CA 90095 USA., Ermolova NV; Department of Neurology and Center for Duchenne Muscular Dystrophy, David Geffen School of Medicine, University of California Los Angeles, 635 Charles Young Dr. South, NRB Room 401, Los Angeles, CA 90095 USA ; Center for Duchenne Muscular Dystrophy at UCLA, Los Angeles, CA 90095 USA., Ishikawa TO; Department of Biological Chemistry, David Geffen School of Medicine, University of California Los Angeles, 341 Boyer Hall, 611 Charles E Young Dr. So, Los Angeles, CA 90095 USA ; Present address: Genomics Business Department, Trans Genic Inc, Kumamoto, 862-0976 Japan., Stout DB; Department of Molecular and Medical Pharmacology, David Geffen School of Medicine, University of California Los Angeles, Los Angeles, CA 90095 USA., Herschman HR; Department of Biological Chemistry, David Geffen School of Medicine, University of California Los Angeles, 341 Boyer Hall, 611 Charles E Young Dr. So, Los Angeles, CA 90095 USA ; Department of Molecular and Medical Pharmacology, David Geffen School of Medicine, University of California Los Angeles, Los Angeles, CA 90095 USA ; Molecular Biology Institute, UCLA, Los Angeles, CA 90095 USA., Spencer MJ; Department of Neurology and Center for Duchenne Muscular Dystrophy, David Geffen School of Medicine, University of California Los Angeles, 635 Charles Young Dr. South, NRB Room 401, Los Angeles, CA 90095 USA ; Center for Duchenne Muscular Dystrophy at UCLA, Los Angeles, CA 90095 USA ; Molecular Biology Institute, UCLA, Los Angeles, CA 90095 USA.

Publikováno v: Skeletal muscle [Skelet Muscle] 2015 Apr 30; Vol. 5, pp. 15. Date of Electronic Publication: 2015 Apr 30 (Print Publication: 2015).

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Akademický článek

Long-term administration of the TNF blocking drug Remicade (cV1q) to mdx mice reduces skeletal and cardiac muscle fibrosis, but negatively impacts cardiac function.

Autor: Ermolova NV; Department of Neurology, David Geffen School of Medicine, University of California, Los Angeles, CA 90095, USA; Center for Duchenne Muscular Dystrophy at UCLA, Los Angeles, CA 90095, USA., Martinez L; Department of Neurology, David Geffen School of Medicine, University of California, Los Angeles, CA 90095, USA; Center for Duchenne Muscular Dystrophy at UCLA, Los Angeles, CA 90095, USA., Vetrone SA; Department of Neurology, David Geffen School of Medicine, University of California, Los Angeles, CA 90095, USA; Center for Duchenne Muscular Dystrophy at UCLA, Los Angeles, CA 90095, USA., Jordan MC; Department of Physiology, David Geffen School of Medicine, University of California, Los Angeles, CA 90095, USA., Roos KP; Department of Physiology, David Geffen School of Medicine, University of California, Los Angeles, CA 90095, USA., Sweeney HL; Department of Physiology, University of Pennsylvania, Philadelphia, PA 19104, USA; Paul Wellstone Muscular Dystrophy Center, Philadelphia, PA 19104, USA., Spencer MJ; Department of Neurology, David Geffen School of Medicine, University of California, Los Angeles, CA 90095, USA; Center for Duchenne Muscular Dystrophy at UCLA, Los Angeles, CA 90095, USA; Paul Wellstone Muscular Dystrophy Center, Philadelphia, PA 19104, USA. Electronic address: mspencer@mednet.ucla.edu.

Publikováno v: Neuromuscular disorders : NMD [Neuromuscul Disord] 2014 Jul; Vol. 24 (7), pp. 583-95. Date of Electronic Publication: 2014 Apr 26.

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