公开(公告)号：WO1998017321A1

公开(公告)日：1998-04-30

申请号：PCT/US1997018611

申请日：1997-10-16

Applicant: VANDERBILT UNIVERSITY ,  BRIGHAM, Kenneth ,  CANONICO, Angelo ,  MEYRICK, Barbara ,  STECENKO, Arlene

Inventor： VANDERBILT UNIVERSITY

IPC: A61K48/00

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 alpha 1 antitrypsin (AAT) related disorders such as respiratory syncytial virus (RSV) infection.

Abstract translation: 本发明提供了用于通过使用常规直接蛋白递送技术目前无法接近的区域中的基因递送和表达的新方法。 特别地，本文提供的方法和相关产品可用于治疗α1抗胰蛋白酶（AAT）相关疾病如呼吸道合胞病毒（RSV）感染。

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：WO02058627A3

公开(公告)日：2003-05-22

申请号：PCT/US0149958

申请日：2001-11-09

Applicant: UNIV VANDERBILT ,  STECENKO ARLENE ,  BRIGHAM KENNETH

Inventor： STECENKO ARLENE ,  BRIGHAM KENNETH

IPC: A61K38/12 ,  A61P13/10 ,  C07K14/47 ,  A61K38/16 ,  C07K14/705 ,  C12N15/62

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：WO1998012312A1

公开(公告)日：1998-03-26

申请号：PCT/US1997016628

申请日：1997-09-18

Applicant: VANDERBILT UNIVERSITY ,  WRIGHT STATE UNIVERSITY ,  STECENKO, Arlene ,  BERNSTEIN, Jack, M.

Inventor： VANDERBILT UNIVERSITY ,  WRIGHT STATE UNIVERSITY

IPC: C12N15/00

CPC classification number: C12N15/1131 ,  A61K38/00 ,  C12N2310/111 ,  Y02A50/463

Abstract: A vector and methods of inhibiting replication of an RNA virus which replicates in the cytoplasm of a cell in a subject, comprising administering to the cell of the subject a composition comprising a vector comprising a promoter functionally linked to a nucleic acid comprising a viral gene, wherein the nucleic acid is in antisense orientation relative to the promoter and wherein the cell is capable of promoting expression from the promoter, thereby transcribing the nucleic acid and inhibiting replication of the RNA virus in the subject. In particular, provided are vectors and methods for treating a respiratory syncytial virus infection.

Abstract translation: 一种载体和抑制在受试者的细胞的细胞质中复制的RNA病毒的复制的方法，包括向受试者的细胞施用包含与包含病毒基因的核酸功能性连接的启动子的载体的组合物， 其中所述核酸相对于启动子处于反义方向，并且其中所述细胞能够促进来自所述启动子的表达，从而转录所述核酸并抑制所述受试者中RNA病毒的复制。 特别地，提供了用于治疗呼吸道合胞病毒感染的载体和方法。