A - Human Necessities – 61 – K
Patent
A - Human Necessities
61
K
A61K 9/14 (2006.01) A01N 43/04 (2006.01) A01N 63/02 (2006.01) C12N 5/00 (2006.01)
Patent
CA 2637678
The present invention provides nanotransporters and delivery complexes for use in delivery of nucleic acid molecules and/or other pharmaceutical agents in vivo and in vitro. In addition, the invention features chemically-modified RNA silencing agents (e.g., siRNAs) that are stable in vivo and silence target RNA that is associated with a metabolic disorder, as well as delivery complexes comprising said RNA silencing agents in association with the nanotransporters of the invention. The featured RNA silencing agents and delivery complexes are effective therapeutics for targeting disease genes, e.g., genes involved in metabolic disorders.
La présente invention concerne des nanotransporteurs et des complexes de délivrance destinés à être utilisés pour délivrer des molécules d'acides nucléiques et/ou d'autres agents pharmaceutiques in vivo et in vitro. En outre, l'invention concerne des agents de RNA silencing modifiés chimiquement (par exemple des ARNsi) qui sont stables in vivo et qui réduisent au silence un ARN cible qui est associé à un trouble métabolique, ainsi que des complexes de délivrance comprenant lesdits agents de RNA silencing associés aux nanotransporteurs de l'invention. Les agents de RNA silencing et les complexes de délivrance présentés sont des agents thérapeutiques efficaces pour cibler des gènes de maladies, par exemple des gènes impliqués dans des troubles métaboliques.
Ogilvy Renault Llp/s.e.n.c.r.l.,s.r.l.
University Of Massachusetts
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