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Motor neuron disease. SMN2 splicing modifiers improve motor function and longevity in mice with spinal muscular atrophy.
Naryshkin, Nikolai A; Weetall, Marla; Dakka, Amal; Narasimhan, Jana; Zhao, Xin; Feng, Zhihua; Ling, Karen K Y; Karp, Gary M; Qi, Hongyan; Woll, Matthew G; Chen, Guangming; Zhang, Nanjing; Gabbeta, Vijayalakshmi; Vazirani, Priya; Bhattacharyya, Anuradha; Furia, Bansri; Risher, Nicole; Sheedy, Josephine; Kong, Ronald; Ma, Jiyuan; Turpoff, Anthony; Lee, Chang-Sun; Zhang, Xiaoyan; Moon, Young-Choon; Trifillis, Panayiota; Welch, Ellen M; Colacino, Joseph M; Babiak, John; Almstead, Neil G; Peltz, Stuart W; Eng, Loren A; Chen, Karen S; Mull, Jesse L; Lynes, Maureen S; Rubin, Lee L; Fontoura, Paulo; Santarelli, Luca; Haehnke, Daniel; McCarthy, Kathleen D; Schmucki, Roland; Ebeling, Martin; Sivaramakrishnan, Manaswini; Ko, Chien-Ping; Paushkin, Sergey V; Ratni, Hasane; Gerlach, Irene; Ghosh, Anirvan; Metzger, Friedrich.
  • Naryshkin NA; PTC Therapeutics, 100 Corporate Court, South Plainfield, NJ 07080, USA.
  • Weetall M; PTC Therapeutics, 100 Corporate Court, South Plainfield, NJ 07080, USA.
  • Dakka A; PTC Therapeutics, 100 Corporate Court, South Plainfield, NJ 07080, USA.
  • Narasimhan J; PTC Therapeutics, 100 Corporate Court, South Plainfield, NJ 07080, USA.
  • Zhao X; PTC Therapeutics, 100 Corporate Court, South Plainfield, NJ 07080, USA.
  • Feng Z; Section of Neurobiology, Department of Biological Sciences, University of Southern California, Los Angeles, CA 90089, USA.
  • Ling KK; Section of Neurobiology, Department of Biological Sciences, University of Southern California, Los Angeles, CA 90089, USA.
  • Karp GM; PTC Therapeutics, 100 Corporate Court, South Plainfield, NJ 07080, USA.
  • Qi H; PTC Therapeutics, 100 Corporate Court, South Plainfield, NJ 07080, USA.
  • Woll MG; PTC Therapeutics, 100 Corporate Court, South Plainfield, NJ 07080, USA.
  • Chen G; PTC Therapeutics, 100 Corporate Court, South Plainfield, NJ 07080, USA.
  • Zhang N; PTC Therapeutics, 100 Corporate Court, South Plainfield, NJ 07080, USA.
  • Gabbeta V; PTC Therapeutics, 100 Corporate Court, South Plainfield, NJ 07080, USA.
  • Vazirani P; PTC Therapeutics, 100 Corporate Court, South Plainfield, NJ 07080, USA.
  • Bhattacharyya A; PTC Therapeutics, 100 Corporate Court, South Plainfield, NJ 07080, USA.
  • Furia B; PTC Therapeutics, 100 Corporate Court, South Plainfield, NJ 07080, USA.
  • Risher N; PTC Therapeutics, 100 Corporate Court, South Plainfield, NJ 07080, USA.
  • Sheedy J; PTC Therapeutics, 100 Corporate Court, South Plainfield, NJ 07080, USA.
  • Kong R; PTC Therapeutics, 100 Corporate Court, South Plainfield, NJ 07080, USA.
  • Ma J; PTC Therapeutics, 100 Corporate Court, South Plainfield, NJ 07080, USA.
  • Turpoff A; PTC Therapeutics, 100 Corporate Court, South Plainfield, NJ 07080, USA.
  • Lee CS; PTC Therapeutics, 100 Corporate Court, South Plainfield, NJ 07080, USA.
  • Zhang X; PTC Therapeutics, 100 Corporate Court, South Plainfield, NJ 07080, USA.
  • Moon YC; PTC Therapeutics, 100 Corporate Court, South Plainfield, NJ 07080, USA.
  • Trifillis P; PTC Therapeutics, 100 Corporate Court, South Plainfield, NJ 07080, USA.
  • Welch EM; PTC Therapeutics, 100 Corporate Court, South Plainfield, NJ 07080, USA.
  • Colacino JM; PTC Therapeutics, 100 Corporate Court, South Plainfield, NJ 07080, USA.
  • Babiak J; PTC Therapeutics, 100 Corporate Court, South Plainfield, NJ 07080, USA.
  • Almstead NG; PTC Therapeutics, 100 Corporate Court, South Plainfield, NJ 07080, USA.
  • Peltz SW; PTC Therapeutics, 100 Corporate Court, South Plainfield, NJ 07080, USA. friedrich.metzger@roche.com speltz@ptcbio.com.
  • Eng LA; SMA Foundation, 888 Seventh Avenue, Suite 400, New York, NY 10019, USA.
  • Chen KS; SMA Foundation, 888 Seventh Avenue, Suite 400, New York, NY 10019, USA.
  • Mull JL; Department of Stem Cell and Regenerative Biology and the Harvard Stem Cell Institute, Harvard University, Cambridge, MA 02138, USA.
  • Lynes MS; Department of Stem Cell and Regenerative Biology and the Harvard Stem Cell Institute, Harvard University, Cambridge, MA 02138, USA.
  • Rubin LL; Department of Stem Cell and Regenerative Biology and the Harvard Stem Cell Institute, Harvard University, Cambridge, MA 02138, USA.
  • Fontoura P; Roche Pharmaceutical Research and Early Development, Roche Innovation Center Basel, F. Hoffmann-La Roche, Grenzacherstrasse 124, 4070 Basel, Switzerland.
  • Santarelli L; Roche Pharmaceutical Research and Early Development, Roche Innovation Center Basel, F. Hoffmann-La Roche, Grenzacherstrasse 124, 4070 Basel, Switzerland.
  • Haehnke D; Roche Pharmaceutical Research and Early Development, Roche Innovation Center Basel, F. Hoffmann-La Roche, Grenzacherstrasse 124, 4070 Basel, Switzerland.
  • McCarthy KD; SMA Foundation, 888 Seventh Avenue, Suite 400, New York, NY 10019, USA.
  • Schmucki R; Roche Pharmaceutical Research and Early Development, Roche Innovation Center Basel, F. Hoffmann-La Roche, Grenzacherstrasse 124, 4070 Basel, Switzerland.
  • Ebeling M; Roche Pharmaceutical Research and Early Development, Roche Innovation Center Basel, F. Hoffmann-La Roche, Grenzacherstrasse 124, 4070 Basel, Switzerland.
  • Sivaramakrishnan M; Roche Pharmaceutical Research and Early Development, Roche Innovation Center Basel, F. Hoffmann-La Roche, Grenzacherstrasse 124, 4070 Basel, Switzerland.
  • Ko CP; Section of Neurobiology, Department of Biological Sciences, University of Southern California, Los Angeles, CA 90089, USA.
  • Paushkin SV; SMA Foundation, 888 Seventh Avenue, Suite 400, New York, NY 10019, USA.
  • Ratni H; Roche Pharmaceutical Research and Early Development, Roche Innovation Center Basel, F. Hoffmann-La Roche, Grenzacherstrasse 124, 4070 Basel, Switzerland.
  • Gerlach I; Roche Pharmaceutical Research and Early Development, Roche Innovation Center Basel, F. Hoffmann-La Roche, Grenzacherstrasse 124, 4070 Basel, Switzerland.
  • Ghosh A; Roche Pharmaceutical Research and Early Development, Roche Innovation Center Basel, F. Hoffmann-La Roche, Grenzacherstrasse 124, 4070 Basel, Switzerland.
  • Metzger F; Roche Pharmaceutical Research and Early Development, Roche Innovation Center Basel, F. Hoffmann-La Roche, Grenzacherstrasse 124, 4070 Basel, Switzerland. friedrich.metzger@roche.com speltz@ptcbio.com.
Science ; 345(6197): 688-93, 2014 Aug 08.
Article en En | MEDLINE | ID: mdl-25104390

Texto completo: 1 Banco de datos: MEDLINE Asunto principal: Pirimidinonas / Atrofia Muscular Espinal / Empalme Alternativo / Cumarinas / Isocumarinas / Bibliotecas de Moléculas Pequeñas / Proteína 2 para la Supervivencia de la Neurona Motora / Longevidad Límite: Animals / Humans Idioma: En Año: 2014 Tipo del documento: Article

Texto completo: 1 Banco de datos: MEDLINE Asunto principal: Pirimidinonas / Atrofia Muscular Espinal / Empalme Alternativo / Cumarinas / Isocumarinas / Bibliotecas de Moléculas Pequeñas / Proteína 2 para la Supervivencia de la Neurona Motora / Longevidad Límite: Animals / Humans Idioma: En Año: 2014 Tipo del documento: Article