Methods and means for efficient skipping of exon 45 in Duchenne muscular dystrophy pre-mRNA


Ontology type: sgo:Patent     


Patent Info

DATE

2018-03-27T00:00

AUTHORS

Josephus Johannes De Kimpe , Gerardus Johannes Platenburg , Judith Christina Theodora Van Deutekom , Annemieke Aartsma-Rus , Garrit-Jan Boudewijn van Ommen

ABSTRACT

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 an isolated muscle cell with a molecule that binds to a continuous stretch of at least 21 nucleotides within said exon. The invention further relates to such molecule used in the method. More... »

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