公开(公告)号：US20170218037A1

公开(公告)日：2017-08-03

申请号：US15328833

申请日：2015-07-24

Applicant: CONSIGLIO NAZIONALE DELLE RICERCHE

Inventor： Claudio PASSANANTI ,  Nicoletta CORBI ,  Maria Grazia DI CERTO ,  Elisabetta MATTEI ,  Cinzia PISANI ,  Georgios STRIMPAKOS ,  Siro LUVISETTO

IPC: C07K14/47 ,  A61K38/17 ,  A61K48/00

CPC classification number: C07K14/4705 ,  A61K38/1709 ,  A61K48/0058 ,  C07K14/4708 ,  C07K2319/81 ,  C12N15/62 ,  C12N2750/14143 ,  C12N2830/002

Abstract: The present invention features modified human transcription factors capable of increasing utrophin expression, recombinant adeno-associated vectors for delivery of the modified human transcription factors, and methods of treating muscle diseases, including Duchenne's muscular dystrophy.

公开(公告)号：US20160303255A1

公开(公告)日：2016-10-20

申请号：US14909854

申请日：2014-07-26

Applicant: CONSIGLIO NAZIONALE DELLE RICERCHE

Inventor： Claudio PASSANANTI ,  Nicoletta CORBI ,  Maria Grazia DI CERTO ,  Elisabetta MATTEI ,  Cinzia PISANI ,  Georgios STRIMPAKOS

IPC: A61K48/00 ,  C07K14/47 ,  C12N15/86 ,  C07K14/005

CPC classification number: A61K48/0058 ,  A61K38/00 ,  C07K14/005 ,  C07K14/4702 ,  C07K14/4708 ,  C07K2319/81 ,  C12N15/86 ,  C12N2710/16632 ,  C12N2750/14143 ,  C12N2750/14171 ,  C12N2830/008 ,  C12N2830/34 ,  G01N33/6893 ,  G01N2800/2878

Abstract: The present invention features recombinant adeno-associated vectors for delivery of genes to both skeletal and cardiac muscle and methods for treatment of muscle defects, including Duchenne's muscular dystrophy.

Abstract translation: 本发明具有用于将基因递送至骨骼肌和心肌的重组腺相关载体和用于治疗肌肉缺陷的方法，包括杜烯氏肌营养不良症。