ACADEMISCH ZIEKENHUIS LEIDEN H.O.D.N. LEIDS UNIVERSITAIR MEDISCH CENTRUM
发明人:
Jan Wijnholds,Lucie Pierrette Francoise Pellissier
申请号：
US14910302
公开号：
US20160194374A1
申请日:
2014.08.05
申请国别(地区):
US
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 CRB1 or CRB3 that resemble CRB2.
