Safety and Proof-of-Concept Study of Oral QLT091001 in Retinitis Pigmentosa Due to Inherited Deficiencies of Retinal Pigment Epithelial 65 Protein (RPE65) or Lecithin:Retinol Acyltransferase (LRAT)
口服 QLT091001 治疗由视网膜色素上皮 65 蛋白 (RPE65) 或卵磷脂：视黄醇酰基转移酶 (LRAT) 遗传性缺陷引起的视网膜色素变性的安全性和概念验证研究
期刊:PLoS One
影响因子:2.6
doi:10.1371/journal.pone.0143846
Scholl, Hendrik P N; Moore, Anthony T; Koenekoop, Robert K; Wen, Yuquan; Fishman, Gerald A; van den Born, L Ingeborgh; Bittner, Ava; Bowles, Kristen; Fletcher, Emily C; Collison, Frederick T; Dagnelie, Gislin; Degli Eposti, Simona; Michaelides, Michel; Saperstein, David A; Schuchard, Ronald A; Barnes, Claire; Zein, Wadih; Zobor, Ditta; Birch, David G; Mendola, Janine D; Zrenner, Eberhart
