Джаскула-Ранга, Винод,Зэк, Дональд,Банз, Фред,Велсби, Дерек
申请号：
EA201990212
公开号：
EA201990212A1
申请日:
2017.07.05
申请国别(地区):
EA
年份:
2020
代理人:
摘要：
Described herein are methods for treating a retinal degeneration in a subject, such as Leber's congenital amaurosis (LCA), retinitis pigmentosa (RP), and glaucoma. Also provided herein are methods of altering expression of one or more gene products in a cell, such as a retinal ganglion cell. Such methods may comprise utilizing a modified nuclease system, such as Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR) system comprising a bidirectional HI promoter and gRNAs directed to retinal degeneration related genes, packaged in a single, compact adeno-associated virus (AAV) particle.
