Title:
【発明の名称】ミニアデノウイルスベクター
Document Type and Number:
Japanese Patent JP2001502883
Kind Code:
A
Abstract:
This invention is related to adenoviral (Ad) vectors and their applications in the field of genetic medicine, including gene transfer, gene therapy, and gene vaccination. More specifically, this invention is related to the Ad vectors that carry the minimal cis-element of the Ad genome (mini-Ad vector) and are capable of delivering transgenes and/or heterologous DNA up to 36 kb. The generation and propagation of the mini-Ad vectors require trans-complementation of a packaging-attenuated and replication-defective helper Ad (helper) in an Ad helper cell line. This invention further comprises a methodology for generating a mini-adenoviral (mini-Ad) vector for use in gene therapy of hemophilia and animal test systems for in vivo evaluation of the Ad vectors. More specifically, this invention describes factor VIII (FVIII) Ad vectors that only contain minimal cis-element of the Ad genome (so-called mini-Ad) and comprise a human FVIII cDNA with other supporting DNA elements up to 36 kb. The FVIII mini-Ad can be generated and preferentially amplified through the assistance of a packaging-attenuated helper Ad and a helper cell line. This invention also reports designs and methods for producing transgenic mouse models that can be used for in vivo testing the mini-Ad.
Inventors:
Zan, way-way
Alemany, Ramon
Die, ifan
Josephus, Stephen
Barragh, Christina
Ayares, David
Schneiderman, Richard
Alemany, Ramon
Die, ifan
Josephus, Stephen
Barragh, Christina
Ayares, David
Schneiderman, Richard
Application Number:
JP54309597A
Publication Date:
March 06, 2001
Filing Date:
May 30, 1997
Export Citation:
Assignee:
Baxter International Inc.
International Classes:
A01K67/027; A61K31/711; A61K35/76; A61K38/43; A61K48/00; A61P7/04; C07K14/755; C12N5/10; C12N7/00; C12N15/09; C12N15/34; C12N15/85; C12N15/861; C12Q1/02; C12Q1/68; G01N21/78; G01N33/53; G01N33/68; A61K38/00; (IPC1-7): C12N15/09; A01K67/027; A61K35/76; A61K38/43; A61K48/00; C12N5/10; C12N7/00; C12Q1/68
Attorney, Agent or Firm:
Hidekazu Miyoshi (1 outside)