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Dr. Brown's laboratory has focused on the identification of gene defects that elucidate the molecular pathogenesis of selected neuromuscular diseases including amyotrophic lateral sclerosis (ALS, also known as Lou Gehrig's disease), muscular dystrophy, adrenoleukodystrophy, hereditary neuropathy and hyperkalemic periodic paralysis. Knowledge of theses disease genes has facilitated the creation of mouse and cell-based models of these disorders. In turn, these resources have allowed study of therapeutic strategies using conventional small molecule approaches and new modalities such as inhibitory RNAi.
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论文共 710 篇作者统计合作学者相似作者
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PLoS biologyno. 1 (2024): e3002462-e3002462
Neurobiology of disease (2024): 106414-106414
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Handbook of Clinical Neurologypp.203-229, (2023)
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Terence R. Flotte,Oguz Cataltepe,Heather L. Gray-Edwards,Allison M. Keeler-Klunk,Rrita Daci, Brittany Owusu-Adjei, Rebecca Artinian, Shestruma Parajuli, Danielle Kokoski,Meghan Blackwood,Toloo Taghian, Mohammed S. Shazeeb,
MOLECULAR THERAPYno. 4 (2023): 6-7
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Nathan Staff,Jonathan Katz,Stacy Lindborg, Jenny Li,Namita Goyal, Robert Miller,Matthew Burford, Kirk Taylor,Yael Gothelf,Yossef Levy,James Berry,Katharine Nicholson,
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JCI INSIGHTno. 9 (2023)
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