Double-stranded rna structures and constructs, and methods...

Details Double-stranded rna structures and constructs, and methods... Double-stranded rna structures and constructs, and methods...

C - Chemistry, Metallurgy

12

N

C12N 15/11 (2006.01) A61K 31/7088 (2006.01) A61K 31/7105 (2006.01) A61K 48/00 (2006.01) A61P 31/00 (2006.01) C07H 21/02 (2006.01) C12P 19/34 (2006.01)

Patent

CA 2502649

The present invention relates to novel double-stranded RNA (dsRNA) structures and dsRNA expression constructs, methods for generating them, and methods of utilizing them for silencing genes. Desirably, these methods specifically inhibit the expression of one or more target genes in a cell or animal (e.g., a mammal such as a human) without inducing toxicity. These methods can be used to prevent or treat a disease or infection by silencing a gene associated with the disease or infection. The invention also provides methods for identifying nucleic acid sequences that modulate a detectable phenotype, such as the function of a cell, the expression of a gene, or the biological activity of a target polypeptide.

L'invention concerne de nouvelles structures d'ARN à double brin (ARNdb) et des constructions d'expression d'ARNdb, des procédés pour les générer, ainsi que des procédés pour les utiliser aux fins du silençage de gènes. De préférence, ces procédés inhibent spécifiquement l'expression d'un ou de plusieurs gènes cibles dans une cellule ou chez un animal (par exemple, un mammifère tel un humain) sans induire de toxicité. Ces procédés peuvent être utilisés pour empêcher ou traiter une maladie ou une infection par silençage d'un gène associé à cette maladie ou à cette infection. L'invention concerne également des procédés servant à identifier des séquences d'acides nucléiques qui modulent un phénotype détectable, comme la fonction d'une cellule, l'expression d'un gène ou l'activité biologique d'un polypeptide cible.

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