公开(公告)号：WO2002058627A2

公开(公告)日：2002-08-01

申请号：PCT/US2001/049958

申请日：2001-11-09

Applicant: VANDERBILT UNIVERSITY ,  STECENKO, Arlene ,  BRIGHAM, Kenneth

Inventor： STECENKO, Arlene ,  BRIGHAM, Kenneth

IPC: A61K

CPC classification number: C07K14/4712 ,  A61K38/00 ,  C07K2319/02

Abstract: The present invention provides a method of treating cystic fibrosis in a human subject diagnosed with cystic fibrosis, comprising administering to the subject, in a pharmaceutically acceptable carrier, an effective amount of a fusion protein, comprising a cystic fibrosis transmembrane conductance regulator (CFTR) and a membrane translocation sequence (MST), whereby the fusion protein can be taken up by affected cells in the subject, thereby treating cystic fibrosis. The present invention also provides a fusion protein comprising a cystic fibrosis transmembrane conductance regulator and a membrane translocation sequence.

Abstract translation: 本发明提供了一种治疗患有囊性纤维化的人类受试者的囊性纤维化的方法，包括在药学上可接受的载体中向受试者施用有效量的融合蛋白，其包含囊性纤维化跨膜传导调节因子（CFTR）和 膜移位序列（MST），由此融合蛋白可被受试者受影响的细胞摄取，从而治疗囊性纤维化。 本发明还提供了包含囊性纤维化跨膜传导调节剂和膜易位序列的融合蛋白。

公开(公告)号：EP0939656A1

公开(公告)日：1999-09-08

申请号：EP97911736.0

申请日：1997-10-16

Applicant: VANDERBILT UNIVERSITY

Inventor： BRIGHAM, Kenneth ,  CANONICO, Angelo ,  MEYRICK, Barbara ,  STECENKO, Arlene

IPC: A61K48 ,  A61K31 ,  A61K38 ,  A61P11 ,  A61P31 ,  A61P43

CPC classification number: A61K38/57 ,  A61K9/1272 ,  A61K38/44 ,  A61K48/00

Abstract: The present invention provides novel methods for gene delivery and expression in areas that are currently inaccessible through the use of conventional direct protein delivery techniques. In particular, the methods and related products provided herein can be used in the treatment of α1 antitrypsin (AAT) related disorders such as respiratory syncytial virus (RSV) infection.