Title:
【発明の名称】治療分子の細胞内送達のためのカチオン性両親媒性化合物およびプラスミド
Document Type and Number:
Japanese Patent JPH10510813
Kind Code:
A
Abstract:
Novel cationic amphiphiles are provided that facilitate transport of biologically active (therapeutic) molecules into cells. The amphiphiles contain lipophilic groups derived from steroids, from mono or dialkylamines, or from alkyl or acyl groups; and cationic groups, protonatable at physiological pH, derived from amines, alkylamines or polyalkylamines. There are provided also therapeutic compositions prepared typically by contacting a dispersion of one or more cationic amphiphiles with the therapeutic molecules. Therapeutic molecules that can be delivered into cells according to the practice of the invention include DNA, RNA, and polypeptides. Representative uses of the therapeutic compositions of the invention include providing gene therapy, and delivery of antisense polynucleotides or biologically active polypeptides to cells. With respect to therapeutic compositions for gene therapy, the DNA is provided typically in the form of a plasmid for complexing with the cationic amphiphile. Novel and highly effective plasmid constructs are also disclosed, including those that are particularly effective at providing gene therapy for clinical conditions complicated by inflammation. Additionally, targeting of organs for gene therapy by intravenous administration of therapeutic compositions is described.
Inventors:
Siegel, Craig S
Harris, David Jay
Lee, Edward Earl
Hubbard, Shirley Sea
Jung, Song H
Eastman, Simon Jay
Marshall, John
Surreal, Ronald Kay
You, Nelson S
Rain, Massille Bee
Lowe, Eric A
Bagley, Rebecca Gee
Chan, Cho-Dan
Harris, David Jay
Lee, Edward Earl
Hubbard, Shirley Sea
Jung, Song H
Eastman, Simon Jay
Marshall, John
Surreal, Ronald Kay
You, Nelson S
Rain, Massille Bee
Lowe, Eric A
Bagley, Rebecca Gee
Chan, Cho-Dan
Application Number:
JP51923696A
Publication Date:
October 20, 1998
Filing Date:
December 08, 1995
Export Citation:
Assignee:
Genzyme Corporation
International Classes:
A61K9/127; A61K47/28; A61K47/48; A61K48/00; A61P29/00; A61P43/00; C07C211/13; C07C217/28; C07C237/06; C12N15/09; C07C271/20; C07C271/34; C07C275/14; C07C279/12; C07C323/25; C07C327/42; C07C333/04; C07J41/00; C07J43/00; C12N15/88; (IPC1-7): C07C211/13; A61K9/127; A61K47/28; A61K48/00; C07C237/06; C07C271/34; C07C279/12; C07J41/00; C12N15/09
Attorney, Agent or Firm:
Aoyama Ryo (1 person outside)
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