作者：
Nikolai A,Naryshkin [1]
;
Marla,Weetall [1]
;
Amal,Dakka [1]
;
Jana,Narasimhan [1]
;
Xin,Zhao [1]
;
Zhihua,Feng [2]
;
Karen K Y,Ling [2]
;
Gary M,Karp [1]
;
Hongyan,Qi [1]
;
Matthew G,Woll [1]
;
Guangming,Chen [1]
;
Nanjing,Zhang [1]
;
Vijayalakshmi,Gabbeta [1]
;
Priya,Vazirani [1]
;
Anuradha,Bhattacharyya [1]
;
Bansri,Furia [1]
;
Nicole,Risher [1]
;
Josephine,Sheedy [1]
;
Ronald,Kong [1]
;
Jiyuan,Ma [1]
;
Anthony,Turpoff [1]
;
Chang-Sun,Lee [1]
;
Xiaoyan,Zhang [1]
;
Young-Choon,Moon [1]
;
Panayiota,Trifillis [1]
;
Ellen M,Welch [1]
;
Joseph M,Colacino [1]
;
John,Babiak [1]
;
Neil G,Almstead [1]
;
Stuart W,Peltz [3]
;
Loren A,Eng [4]
;
Karen S,Chen [4]
;
Jesse L,Mull [5]
;
Maureen S,Lynes [5]
;
Lee L,Rubin [5]
;
Paulo,Fontoura [6]
;
Luca,Santarelli [6]
;
Daniel,Haehnke [6]
;
Kathleen D,McCarthy [4]
;
Roland,Schmucki [6]
;
Martin,Ebeling [6]
;
Manaswini,Sivaramakrishnan [6]
;
Chien-Ping,Ko [2]
;
Sergey V,Paushkin [4]
;
Hasane,Ratni [6]
;
Irene,Gerlach [6]
;
Anirvan,Ghosh [6]
;
Friedrich,Metzger [7]
作者单位：
PTC Therapeutics, 100 Corporate Court, South Plainfield, NJ 07080, USA.
[1]
Section of Neurobiology, Department of Biological Sciences, University of Southern California, Los Angeles, CA 90089, USA.
[2]
PTC Therapeutics, 100 Corporate Court, South Plainfield, NJ 07080, USA. friedrich.metzger@roche.com speltz@ptcbio.com.
[3]
SMA Foundation, 888 Seventh Avenue, Suite 400, New York, NY 10019, USA.
[4]
Department of Stem Cell and Regenerative Biology and the Harvard Stem Cell Institute, Harvard University, Cambridge, MA 02138, USA.
[5]
Roche Pharmaceutical Research and Early Development, Roche Innovation Center Basel, F. Hoffmann-La Roche, Grenzacherstrasse 124, 4070 Basel, Switzerland.
[6]
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.
[7]
主题词
投药, 口服(Administration, Oral);选择性剪接(Alternative Splicing);动物(Animals);细胞, 培养的(Cells, Cultured);香豆素类(Coumarins);疾病模型, 动物(Disease Models, Animal);药物评价, 临床前(Drug Evaluation, Preclinical);人类(Humans);异香豆素类(Isocoumarins);长寿(Longevity);小鼠(Mice);肌萎缩, 脊髓性(Muscular Atrophy, Spinal);嘧啶酮类(Pyrimidinones);RNA, 信使(RNA, Messenger);序列缺失(Sequence Deletion);小分子化合物库(Small Molecule Libraries);运动神经元生存蛋白质2(Survival of Motor Neuron 2 Protein)
DOI
10.1126/science.1250127
PMID
25104390
发布时间
2014-11-17
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