Method for efficient exon (44) skipping in duchenne muscular...

C - Chemistry – Metallurgy – 12 – N

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C12N 15/11 (2006.01) A61K 31/7088 (2006.01) C12N 15/86 (2006.01)

Patent

CA 2723968

The invention relates to a nucleic acid molecule that binds and/or is complementary to the nucleotide molecule having sequence 5'-GUGGCUAACAGAAGCU (SEQ ID NO 1) and to its use in a method for inducing skipping of exon 44 of the DMD gene in a DMD patient.

Linvention concerne une molécule dacide nucléique qui se lie et/ou est complémentaire de la molécule de nucléotide ayant la séquence 5-GUGGCUAACAGAAGCU (SEQ ID NO 1) et son utilisation dans un procédé dinduction de lomission de lexon 44 du gène DMD chez un patient DMD.

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