C - Chemistry – Metallurgy – 12 – N
Patent
C - Chemistry, Metallurgy
12
N
C12N 15/11 (2006.01) A61K 31/7105 (2006.01)
Patent
CA 2741629
The invention relates to a method for inducing or promoting skipping of exon 45 of DMD pre-mRNA in a Duchenne Muscular Dystrophy patient, preferably in an isolated (muscle) cell, the method comprising providing said cell with an antisense molecule that binds to a continuous stretch of at least 21 nucleotides within said exon. The invention further relates to such antisense molecule used in said method.
La présente invention concerne un procédé permettant dinduire ou de favoriser le saut de lexon 45 de lARN pré-messager du gène DMD chez un patient atteint dune dystrophie musculaire de Duchenne, de préférence dans une cellule (musculaire) isolée, le procédé consistant à mettre ladite cellule en présence dune molécule antisens qui se lie à une suite continue dau moins 21 nucléotides au sein dudit exon. Linvention concerne en outre ladite molécule antisens utilisée dans ledit procédé.
Aartsma-Rus Annemieke
de Kimpe Josephus Johannes
Platenburg Gerard Johannes
Van Deutekom Judith Christina Theodora
Van Ommen Garrit-Jan Boudewijn
Academisch Ziekenhuis Leiden
Gowling Lafleur Henderson Llp
Prosensa B.v.
Prosensa Holding Bv
Prosensa Technologies B.v.
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