COMPOSITION D'HOMOLOGUES DE CRUMBS ET VAA RECOMBINANTS ET PROCEDES POUR TRAITER LA LCA-8 ET LA RP PROGRESSIVE

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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：

CA2919995

公开号：

CA2919995A1

申请日:

2014.08.05

申请国别(地区):

CA

年份:

2015

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 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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中国工程科技知识中心

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