Burli, Roland Werner,Esmieu, William Rameshchandra Krishna,Lock, Christopher James,Malagu, Karine Fabienne,Owens, Andrew Pate,Harte, William Edward
申请号：
AU2013334139
公开号：
AU2013334139A1
申请日:
2013.10.25
申请国别(地区):
AU
年份:
2015
代理人:
摘要：
Compounds, compositions and methods for the treatment of retinal degenerative diseases, such as retinitis pigmentosa, Lebers congenital Amaurosis, Syndromic retinal degenerations, age-related macular degeneration and Usher Syndrome, and hearing loss associated with Usher Syndrome are described herein. Usher Syndrome, a rare genetic disorder and a leading cause of deafness and blindness, is associated with a mutation in any one of ten genes.
