RECOMBINANT AAV CRUMBS HOMOLOGUE COMPOSITION AND METHODS FOR TREATING LCA 8 AND PROGRESSIVE RP

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专利权人:

发明人:

WIJNHOLDS JAN,PELLISSIER LUCIE PIERRETTE FRAN§OISE

申请号：

IN201627003619

公开号：

IN201627003619A

申请日:

2016.02.01

申请国别(地区):

IN

年份:

2016

代理人:

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 CRB1or CRB3 that resemble CRB2.

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