C - Chemistry – Metallurgy – 12 – N
Patent
C - Chemistry, Metallurgy
12
N
C12N 15/11 (2006.01) A61K 31/7036 (2006.01) A61K 31/7088 (2006.01) A61K 38/00 (2006.01) A61K 45/00 (2006.01) C07H 21/02 (2006.01) C07H 21/04 (2006.01) C12N 15/63 (2006.01) C12Q 1/68 (2006.01) G01N 33/53 (2006.01)
Patent
CA 2664189
Disclosed are compositions and methods related to the interaction of polyCUG and polyCCUG repeat RNA and proteins that bind to these repetitive RNA sequences. Also disclosed are methods of treating DM1 or DM2 comprising inhibiting the interaction of poly(CUG)exp or .rho.oly(CCUG)exp RNA with muscleblind proteins, or by causing improvement of spliceopathy in myotonic dystrophy.
La présente invention concerne des compositions et procédés se rapportant à l'interaction entre polyCUG et ARN à répétition de polyCCUG, ainsi que des protéines qui se lient à ces séquences d'ARN répétitives. L'invention concerne également des procédés permettant de traiter les DM1 et DM2 en inhibant l'interaction de l'ARN à poly(CUG)exp ou à poly(CCUG)exp avec des protéines MuscleBlind, ou en provoquant une amélioration de la souffrance des épissures (spliceopathy) dans la distrophie myotonique.
Miller Jill
Osborne Robert
Sobczak Krzysztof
Swanson Maurice S.
Thornton Charles A.
Gowling Lafleur Henderson Llp
Swanson Maurice S.
University Of Florida Research Foundation Inc.
University Of Rochester
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