C - Chemistry – Metallurgy – 12 – N
Patent
C - Chemistry, Metallurgy
12
N
C12N 15/113 (2010.01) A61K 31/713 (2006.01) C07H 21/02 (2006.01)
Patent
CA 2701845
The invention relates to siRNA compounds possessing novel sequences and structural motifs which down-regulate the expression of specific human genes. The invention also relates to pharmaceutical compositions comprising such compounds and a pharmaceutically acceptable carrier. The present invention also provides a method of treating and/or preventing the incidence or severity of various diseases or conditions associated with the genes and/or symptoms associated with such diseases or conditions comprising administering to a subject in need of treatment for such disease or condition and/or symptom the compound or the pharmaceutical composition in a therapeutically effective dose so as to thereby treat the subject.
L'invention concerne des composés d'ARNsi possédant de nouvelles séquences et des motifs structuraux qui régulent de manière négative l'expression de gènes humains spécifiques. L'invention concerne également des compositions pharmaceutiques comprenant de tels composés et un support pharmaceutiquement acceptable. La présente invention concerne également un procédé de traitement et/ou de prévention de l'incidence ou de la gravité de diverses maladies ou divers états associés aux gènes et/ou de symptômes associés à de telles maladies ou de tels états consistant à administrer à un sujet, lequel a besoin d'un traitement pour une telle maladie ou un tel état et/ou un symptôme, le composé ou la composition pharmaceutique selon une dose thérapeutiquement efficace, et ainsi traiter le sujet.
Aygun Huseyin
Beigelman Leonid
Feinstein Elena
Kalinski Hagar
Mcswiggen James
Norton Rose Or S.e.n.c.r.l. S.r.l./llp
Quark Pharmaceuticals Inc.
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