首页> Molecular therapy : the journal of the American Society of Gene Therapy> The Amyloid Inhibitor CLR01 Relieves Autophagy and Ameliorates Neuropathology in a Severe Lysosomal Storage Disease.

The Amyloid Inhibitor CLR01 Relieves Autophagy and Ameliorates Neuropathology in a Severe Lysosomal Storage Disease.

导出 OA

翻译打印收藏纠错

第一作者： Antonio,Monaco

第一单位： Telethon Institute of Genetics and Medicine, Via Campi Flegrei 34, 80078 Pozzuoli, Naples, Italy.

作者： Antonio,Monaco ^[1] ; Veronica,Maffia ^[1] ; Nicolina Cristina,Sorrentino ^[1] ; Irene,Sambri ^[1] ; Yulia,Ezhova ^[1] ; Teresa,Giuliano ^[1] ; Vincenzo,Cacace ^[1] ; Edoardo,Nusco ^[1] ; Maria,De Risi ^[2] ; Elvira,De Leonibus ^[2] ; Thomas,Schrader ^[3] ; Frank-Gerrit,Klärner ^[3] ; Gal,Bitan ^[4] ; Alessandro,Fraldi ^[5]

作者单位： Telethon Institute of Genetics and Medicine, Via Campi Flegrei 34, 80078 Pozzuoli, Naples, Italy. ^[1] Telethon Institute of Genetics and Medicine, Via Campi Flegrei 34, 80078 Pozzuoli, Naples, Italy; Institute of Cellular Biology and Biochemistry, CNR, Via Ramarini 33, 00015 Monterotondo Scalo, Rome, Italy. ^[2] Department of Chemistry, University of Duisburg-Essen, Universitaetsstrasse 7, 45117 Essen, Germany. ^[3] Department of Neurology, David Geffen School of Medicine, Brain Research Institute, University of California, Los Angeles, 635 Charles E. Young Drive South, Los Angeles, CA 90095-7334, USA; Molecular Biology Institute, University of California, Los Angeles, 635 Charles E. Young Drive South, Los Angeles, CA 90095-7334, USA. ^[4] Telethon Institute of Genetics and Medicine, Via Campi Flegrei 34, 80078 Pozzuoli, Naples, Italy; Department of Translational Medicine, University of Naples "Federico II," Via Sergio Pansini 5, 80131, Naples, Italy. Electronic address: fraldi@tigem.it. ^[5]

关键词 amyloid aggregation autophagy lysosomal storage disease molecular tweezers mucopolysaccharidosis type IIIA