A - Human Necessities – 61 – K
Patent
A - Human Necessities
61
K
A61K 48/00 (2006.01) A61K 31/7125 (2006.01) C07H 21/00 (2006.01) C07H 21/04 (2006.01) C12N 15/11 (2006.01)
Patent
CA 2265917
The invention relates to modified oligonucleotides that are useful for studies of gene expression and for the antisense therapeutic approach. The invention provides modified oligonucleotides that inhibit gene expression and that produce fewer side effects than conventional phosphorothioate oligonucleotides. In particular, the invention provides modified CpG-containing oligonucleotides that result in reduced splenomegaly and platelet depletion when administered to a mammal, relative to conventional CpG-containing phosphorothioate oligonucleotides. The ivnention further provides methods for using such oligonucleotides to modulate gene expression in vivo, including such use for therapeutic treatment of diseases caused by aberrant gene expression.
L'invention concerne des oligonucléotides modifiés utiles pour des études de l'expression génique et pour l'approche thérapeutique par antisens. Elle permet de produire des oligonucléotides modifiés qui inhibent l'expression génétique et ont peu d'effets secondaires en comparaison aux oligonucléotides traditionnels à base de thiophosphate. L'invention propose notamment des oligonucléotides modifiés contenant CpG qui, administrés à un mammifère, permettent de mieux réduire la splénomégalie et l'appauvrissement plaquettaire que les oligonucléotides traditionnels à base de thiophosphate contenant CpG. L'invention propose également des méthodes d'utilisation desdits oligonucléotides qui ont pour but de moduler in vivo l'expression génétique, y compris leur utilisation dans le traitement thérapeutique des maladies provoquées par une expression génétique aberrante.
Hybridon Inc.
Idera Pharmaceuticals Inc.
Mbm Intellectual Property Law Llp
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