Title:
【発明の名称】高脂血症の遺伝子治療のための方法および化合物
Document Type and Number:
Japanese Patent JP2002534353
Kind Code:
A
Abstract:
The present invention concerns compositions and methods for the introduction of specific genetic changes in endogenous genes of the cells of an animal. The genetic changes are effected by oligonucleotides or oligonucleotide derivatives and analogs, which are generally less than about 100 nucleotides in length. The invention provides for macromolecular carriers, optionally incorporating ligands for clathrin coated pit receptors. In one embodiment the ligand is a lactose or galactose and the genetic changes are made in hepatocytes. By means of the invention up to 40% of the copies of a target gene have been changed in vitro. Repair of mutant genes having a Crigler-Najjar like phenotype and Hemophilia B phenotype were observed.
Inventors:
Steer, Clifford, Jay.
Clen, Besty, Tea.
Bandio Pad-Hai, Paramita, Tea.
Roy Choudhry, Janta
Clen, Besty, Tea.
Bandio Pad-Hai, Paramita, Tea.
Roy Choudhry, Janta
Application Number:
JP2000531065A
Publication Date:
October 15, 2002
Filing Date:
August 28, 1998
Export Citation:
Assignee:
Regents of the University of Minnesota
Albert Einstein College of Medicine of Yeshiva University
Albert Einstein College of Medicine of Yeshiva University
International Classes:
C12N15/09; A01N43/04; A61K9/127; A61K9/50; A61K9/51; A61K31/7088; A61K31/7105; A61K31/711; A61K31/712; A61K31/7125; A61K47/24; A61K48/00; A61P3/06; C07H21/00; C07H21/04; C12N15/11; C12N15/85; C12Q1/68; (IPC1-7): A61K48/00; A61K9/50; A61K9/51; A61K31/711; A61K47/24; A61P3/06; C07H21/04; C12N15/09
Attorney, Agent or Firm:
Yusuke Hiraki (1 person outside)