Recombinant AAV-Crumbs homologue composition and methods for treating LCA-8 and progressive RP

• 收藏
• 分享

专利权人:

Academisch Ziekenhuis Leiden H.O.D.N. Leids Universitair Medisch Centrum

发明人:

Wijnholds, Jan,Pellissier, Lucie Pierrette Francoise

申请号：

AU2014305218

公开号：

AU2014305218A1

申请日:

2014.08.05

申请国别(地区):

AU

年份:

2016

代理人:

摘要：

The present invention relates toa Crumbs homologue (CRB) therapeutic for use as a medicament or in a method of treatment or prophylaxis, for example in the treatment or prophylaxis of a retinal disorder due to mutations in the Crumbs homologue-1 (CRB1)gene, such as Lebers congenital amaurosis 8(LCA8) or retinitis pigmentosa 12 (RP12).In particular, the present invention relates to a recombinant viral vector comprising CRB2 or modified non-toxic forms of either CRB1or CRB3 that resemble CRB2.

来源网站：

中国工程科技知识中心

来源网址：

http://www.ckcest.cn/home/