Title:
The use in the expression vector and gene therapy containing a change human U1snRNA molecule, the gene which is coding the change human U1snRNA molecule, and a gene
Document Type and Number:
Japanese Patent JP5985487
Kind Code:
B2
Abstract:
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.
Inventors:
Pagani, Franco
Pinotti, Mirco
Pinotti, Mirco
Application Number:
JP2013533322A
Publication Date:
September 06, 2016
Filing Date:
October 14, 2011
Export Citation:
Assignee:
Universita Delli Studio Di Ferrara
International Classes:
C12N15/113; A61K31/7088; A61K48/00; A61P3/00; A61P7/04; A61P21/00; C12Q1/68
Foreign References:
| US20100087511 |
Other References:
Blood,2009年,Vol.113, No.25,p.6461-6464
The Journal of Biological Chemistry,2000年,Vol.275, No.46,p.35914-35919
Nature Genetics,2002年,Vol.30,p.426-429
The Journal of Biological Chemistry,2000年,Vol.275, No.46,p.35914-35919
Nature Genetics,2002年,Vol.30,p.426-429
Attorney, Agent or Firm:
Kurata Masatoshi
Yoshihiro Fukuhara
Makoto Nakamura
Nobuhisa Nogawa
Toshio Shirane
Takashi Mine
Katsu Sunagawa
Yoshihiro Fukuhara
Makoto Nakamura
Nobuhisa Nogawa
Toshio Shirane
Takashi Mine
Katsu Sunagawa