EP 0979311 A1 20000216 - $i(IN VIVO) USE OF RECOMBINAGENIC OLIGONUCLEOBASES TO CORRECT GENETIC LESIONS IN HEPATOCYTES
Title (en)
$i(IN VIVO) USE OF RECOMBINAGENIC OLIGONUCLEOBASES TO CORRECT GENETIC LESIONS IN HEPATOCYTES
Title (de)
$i(IN VIVO)VERWENDUNG REKOMBINAGENISCHER OLIGONUKLEOBASEN ZUR KORREKTUR GENETISCHER DEFEKTE IN HEPATOZYTEN
Title (fr)
UTILISATION $i(IN VIVO) D'OLIGONUCLEOBASES RECOMBINAGENES POUR CORRIGER LES LESIONS GENETIQUES DES HEPATOCYTES
Publication
Application
Priority
- US 9808834 W 19980430
- US 4528897 P 19970430
- US 5483797 P 19970805
- US 6499697 P 19971110
Abstract (en)
[origin: WO9849350A1] 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 copiies 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.
IPC 1-7
IPC 8 full level
C12N 15/09 (2006.01); A61K 9/107 (2006.01); A61K 9/127 (2006.01); A61K 31/7088 (2006.01); A61K 48/00 (2006.01); A61P 3/10 (2006.01); A61P 7/00 (2006.01); A61P 7/04 (2006.01); C12N 15/10 (2006.01)
CPC (source: EP KR)
A61K 9/1075 (2013.01 - EP); A61K 9/1272 (2013.01 - EP); A61P 3/10 (2017.12 - EP); A61P 7/00 (2017.12 - EP); A61P 7/04 (2017.12 - EP); C12N 15/102 (2013.01 - EP); C12Q 1/68 (2013.01 - KR)
Citation (search report)
See references of WO 9849350A1
Designated contracting state (EPC)
AT BE CH DE DK ES FI FR GB GR IE IT LI LU MC NL PT SE
DOCDB simple family (publication)
WO 9849350 A1 19981105; WO 9849350 A8 20000525; AU 7365498 A 19981124; AU 749410 B2 20020627; CA 2288209 A1 19981105; CN 1268186 A 20000927; EP 0979311 A1 20000216; JP 2002511851 A 20020416; KR 20010020375 A 20010315; NZ 500694 A 20010928
DOCDB simple family (application)
US 9808834 W 19980430; AU 7365498 A 19980430; CA 2288209 A 19980430; CN 98806642 A 19980430; EP 98920930 A 19980430; JP 54742998 A 19980430; KR 19997009997 A 19991029; NZ 50069498 A 19980430