EP 0693130 A1 19960124 - TARGETED DELIVERY OF GENES ENCODING ANTISENSE POLYRIBONUCLEOTIDES
Title (en)
TARGETED DELIVERY OF GENES ENCODING ANTISENSE POLYRIBONUCLEOTIDES
Title (de)
GEZIEHLTE FREISETZUNG VON GENEN DIE FÜR GEGENLAÜFIGE POLYRIBONUKLEOTIDE KODIEREN
Title (fr)
APPORT CIBLE DE GENES CODANT DES POLYRIBONUCLEOTIDES ANTISENS
Publication
Application
Priority
- US 9403643 W 19940404
- US 4294393 A 19930405
Abstract (en)
[origin: WO9423050A1] Molecular complexes for targeting a gene encoding an antisense polyribonucleotide to a specific cell in vivo obtaining production of the polyribonucleotide within the targeted cell, and effecting specific inhibition of the expression of cellular or noncellular genes are disclosed. An expressible gene encoding a desired antisense polyribonucleotide is complexed to a conjugate of a cell-specific binding agent and a gene-binding agent. The cell-specific binding agent is specific for a cellular surface structure which mediates internalization of ligands by endocytosis. An example is the asialoglycoprotein receptor of hepatocytes. The gene-binding agent is a compound such as a polycation which stably complexes the gene under extracellular conditions and releases the gene under intracellular conditions so that it can function within a cell. The molecular complex is stable and soluble in physiological fluids and can be used in antisense gene therapy to selectively transfect cells in vivo or in vitro to provide for production of the antisense polyribonucleotide within the targeted cell, and inhibition of the expression of cellular or noncellular genes.
IPC 1-7
IPC 8 full level
C12N 15/09 (2006.01); A61K 31/70 (2006.01); A61K 47/48 (2006.01); A61K 48/00 (2006.01); A61P 31/00 (2006.01); A61P 31/04 (2006.01); A61P 31/12 (2006.01); C07H 21/04 (2006.01); C12N 9/00 (2006.01); C12N 15/113 (2010.01); C12N 15/87 (2006.01); C12R 1/92 (2006.01)
CPC (source: EP)
A61K 47/645 (2017.07); A61K 48/00 (2013.01); A61P 31/00 (2017.12); A61P 31/04 (2017.12); A61P 31/12 (2017.12); C12N 15/1131 (2013.01); C12N 15/87 (2013.01); C12N 2310/12 (2013.01); C12N 2310/3513 (2013.01)
Citation (search report)
See references of WO 9423050A1
Designated contracting state (EPC)
AT BE CH DE DK ES FR GB GR IE IT LI LU MC NL PT SE
DOCDB simple family (publication)
WO 9423050 A1 19941013; AU 6497894 A 19941024; AU 686614 B2 19980212; CA 2159916 A1 19941013; EP 0693130 A1 19960124; JP H08508645 A 19960917
DOCDB simple family (application)
US 9403643 W 19940404; AU 6497894 A 19940404; CA 2159916 A 19940404; EP 94912389 A 19940404; JP 52243394 A 19940404