Pan Shan-Shan, Zheng Qin-Xiang, Li Wen-Sheng, Pang Ji-Jing
Eye Hospital, Wenzhou Medical College, Wenzhou 325027, China.
Zhonghua Yan Ke Za Zhi. 2011 Jan;47(1):83-7.
Leber congenital amaurosis (LCA) is an early onset retinal dystrophy that causes severe visual impairment. With the development of molecular genetics and the therapeutic gene replacement technology, the adeno-associated viral (AAV) vector-mediated gene therapy for LCA achieved encouraging progress in the past decade. The success of the Phase I clinical trials of human RPE65 gene therapy for LCA II patients makes it a pioneer in the field of retinal gene therapy and brings light to the cure of other hereditary retinopathy. This article briefly reviews the recent developments in the preclinical animal experiments and Phase I clinical trials for LCA.
莱伯先天性黑蒙（LCA）是一种早发性视网膜营养不良，可导致严重视力损害。随着分子遗传学和治疗性基因替代技术的发展，在过去十年中，腺相关病毒（AAV）载体介导的LCA基因治疗取得了令人鼓舞的进展。人类RPE65基因治疗LCA II型患者的I期临床试验的成功使其成为视网膜基因治疗领域的先驱，并为其他遗传性视网膜病变的治疗带来了希望。本文简要回顾了LCA临床前动物实验和I期临床试验的最新进展。
