Hammerhead ribozymes with extended cleavage rule

Details Hammerhead ribozymes with extended cleavage rule Hammerhead ribozymes with extended cleavage rule

C - Chemistry, Metallurgy

12

N

C12N 15/11 (2006.01) A61K 31/70 (2006.01) C07H 21/00 (2006.01) C12N 9/00 (2006.01) A61K 38/00 (2006.01)

Patent

CA 2295207

Disclosed are compositions having an RNA-cleavage activity, as well as their use for cleaving RNA-substrates in vitro and in vivo. The compositions contain an active center, the subnits of which are selected from nucleotides and/or nucleotide analogues, as well as flanking regions contributing to the formation of a specific hybridization with an RNA substrate. Preferred compositions form, in combination with an RNA substrate, a structure resembling a hammerhead structure. The active center of the disclosed compositions is characterized by the presence of I15.1 which allows cleavage of RNA substrates having C16.1.

On décrit des compositions présentant une activité de clivage de l'ARN ainsi que leur utilisation pour couper in vitro et in vivo des substrats d'ARN. Les compositions contiennent un centre actif dont les sous-unités sont sélectionnées parmi des nucléotides et/ou des analogues de nucléotides, ainsi que des régions flanquantes contribuant à la formation d'une hybridation spécifique avec un substrat d'ARN. Les compositions préférées forment, en combinaison avec un substrat d'ARN, une structure qui ressemble à une structure en tête de marteau. Le centre actif des compositions de cette invention se caractérise par la présence de I?15.1¿ qui permet de couper des substrats d'ARN ayant un C?16.1¿.

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