发明公开
EP3030665A1 RECOMBINANT AAV-CRUMBS HOMOLOGUE COMPOSITION AND METHODS FOR TREATING LCA-8 AND PROGRESSIVE RP
审中-公开
重组AAV-CRUMBS同源组合物和治疗LCA-8和进展性RP的方法
- 专利标题： RECOMBINANT AAV-CRUMBS HOMOLOGUE COMPOSITION AND METHODS FOR TREATING LCA-8 AND PROGRESSIVE RP
- 专利标题（中）： 重组AAV-CRUMBS同源组合物和治疗LCA-8和进展性RP的方法
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申请号： EP14761426.7申请日： 2014-08-05
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公开(公告)号： EP3030665A1公开(公告)日： 2016-06-15
- 发明人: WIJNHOLDS, Jan , PELLISSIER, Lucie Pierrette Françoise
- 申请人： Academisch Ziekenhuis Leiden H.O.D.N. Leids Universitair Medisch Centrum
- 申请人地址： Albinusdreef 2 2333 ZA Leiden NL
- 专利权人： Academisch Ziekenhuis Leiden H.O.D.N. Leids Universitair Medisch Centrum
- 当前专利权人： Academisch Ziekenhuis Leiden H.O.D.N. Leids Universitair Medisch Centrum
- 当前专利权人地址： Albinusdreef 2 2333 ZA Leiden NL
- 代理机构： Potter Clarkson LLP
- 优先权： EP13179254 20130805
- 国际公布： WO2015020522 20150212
- 主分类号： C12N15/86
- IPC分类号： C12N15/86 ; A61K48/00 ; C07K14/47
摘要：
The present invention relates to a 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 Leber's 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 CRB1 or CRB3 that resemble CRB2.
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