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1.发明公开A MODIFIED HUMAN U1SNRNA MOLECULE, A GENE ENCODING FOR THE MODIFIED HUMAN U1SNRNA MOLECULE, AN EXPRESSION VECTOR INCLUDING THE GENE, AND THE USE THEREOF IN GENE THERAPY 有权修饰的人U1SNRNA分子，修饰的人U1SNRNA分子编码基因，表达载体;该基因及其用途的基因治疗
公开(公告)号：EP2627339A1
公开(公告)日：2013-08-21
申请号：EP11799816.1
申请日：2011-10-14
发明人： PAGANI, Franco , PINOTTI, Mirko
IPC分类号： A61K31/7105 , C12N15/113 , A61K48/00
CPC分类号： C12N15/113 , C12N15/111 , C12N15/1137 , C12N15/1138 , C12N2310/11 , C12N2320/33 , C12N2330/51 , C12Y304/21022 , C12Y306/03049
摘要： A modified human U1snRNA molecule is described, the target sequence of which is located in a region of the pre-mRNA of the target gene comprised between 2 and 50 base pairs downstream of an exon/intron junction site, which is capable of restoring the correct splicing of a target gene of therapeutic interest bearing a mutation which induces exon skipping and resulting in a genetic disease. Modified human U1snRNA molecules are described by way of example for the correction of diseases associated with exon skipping, such as spinal muscular atrophy, hemophilia B, and cystic fibrosis.
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2.发明授权A MODIFIED HUMAN U1SNRNA MOLECULE, A GENE ENCODING FOR THE MODIFIED HUMAN U1SNRNA MOLECULE, AN EXPRESSION VECTOR INCLUDING THE GENE, AND THE USE THEREOF IN GENE THERAPY 有权修饰的人U1SNRNA分子，修饰的人U1SNRNA分子编码基因，表达载体;该基因及其用途的基因治疗
公开(公告)号：EP2627339B1
公开(公告)日：2015-05-27
申请号：EP11799816.1
申请日：2011-10-14
发明人： PAGANI, Franco , PINOTTI, Mirko
IPC分类号： A61K31/7105 , C12N15/113 , A61K48/00
CPC分类号： C12N15/113 , C12N15/111 , C12N15/1137 , C12N15/1138 , C12N2310/11 , C12N2320/33 , C12N2330/51 , C12Y304/21022 , C12Y306/03049
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