Enhanced antisense oligonucleotides

Details Enhanced antisense oligonucleotides Enhanced antisense oligonucleotides

C - Chemistry, Metallurgy

12

Q

C12Q 1/68 (2006.01) A01N 43/04 (2006.01) C07H 21/02 (2006.01) C07H 21/04 (2006.01) C12N 15/63 (2006.01) C12N 15/88 (2006.01) C12P 19/34 (2006.01)

Patent

CA 2580504

Described herein are gap-widened antisense oligonucleotides having improved therapeutic index as compared to 5-10-5 MOE gapmer antisense oligonucleotides of the same sequence. Also described are methods of reducing a target RNA in an animal using the gap-widened antisense oligonucleotides of the present invention. Further, are methods for selecting a gap-widened antisense oligonucleotides.

L'invention concerne des oligonucléotides antisens élargis par un espace (<= gap >=) à indice thérapeutique amélioré par rapport aux oligonucléotides antisens de type espacemère (<= gapmer >=) 5-10-5 MOE de la même séquence. On décrit aussi des procédés de réduction d'ARN cible chez un animal faisant appel aux oligonucléotides élargis en question, et enfin des procédés de sélection pour ce type d'oligonucléotide.

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