Gene Therapy for Retinal Degeneration

被引：61

作者：

Apte, Rajendra S. ^{[1
]}

机构：

[1] Washington Univ, Sch Med, 660 South Euclid Ave,Box 8096, St Louis, MO 63110 USA

来源：

CELL | 2018年 / 173卷 / 01期

关键词：

D O I：

10.1016/j.cell.2018.03.021

中图分类号：

Q5 [生物化学]; Q7 [分子生物学];

学科分类号：

071010 ; 081704 ;

摘要：

Biallelic mutations in the RPE65 gene are associated with inherited retinal degenerations/dystrophies (IRD) and disrupt the visual cycle, leading to loss of vision. A new adenoviral vector-based gene therapy surgically delivered to retinal cells provides normal human RPE65 protein that can restore the visual cycle and some vision.

引用

页码：5 / 5

页数：1