公开(公告)号：WO1992019730A1

公开(公告)日：1992-11-12

申请号：PCT/US1992002465

申请日：1992-03-27

Applicant: VANDERBILT UNIVERSITY

Inventor： VANDERBILT UNIVERSITY ,  BRIGHAM, Kenneth ,  CONARY, Jon ,  CANONICO, Angelo ,  MEYRICK, Barbara

IPC: C12N15/00

CPC classification number: C12N15/85 ,  C07K14/8125

Abstract: A plasmid consists essentially of a small pCMV4 expression vector including a coding sequence of human alpha-1 antitrypsin incorporated therein. The present invention further provides a method for delivering the gene to a patient, the mechanism including a liposome including the expression plasmid incorporated therein, the plasmid being capable of expression of the gene extrachromosomally in the cells of a target tissue and being unincorporable into the chromosome of the cells of the target tissue. The present invention further provides a primer for inserting the coding sequence of the protein into the expression vector. Finally, the present invention provides a method of treatment for deficiency of a gene product in cells of a target tissue by using the novel liposome.

Abstract translation: 质粒基本上由包含其中结合有人α-1抗胰蛋白酶的编码序列的小pCMV4表达载体组成。 本发明还提供了将基因递送至患者的方法，所述方法包括包含其中并入的表达质粒的脂质体，所述质粒能够在靶组织的细胞中染色体外表达基因，并且不能并入染色体 的靶组织的细胞。 本发明还提供了用于将蛋白质的编码序列插入到表达载体中的引物。 最后，本发明提供了通过使用新的脂质体来治疗目标组织的细胞中的基因产物的缺陷的方法。

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.