A - Human Necessities – 61 – K
Patent
A - Human Necessities
61
K
A61K 31/7105 (2006.01) A61K 48/00 (2006.01) A61P 21/00 (2006.01) C12N 5/10 (2006.01)
Patent
CA 2741793
The invention relates a method wherein a molecule is used for inducing and/or promoting skipping of at least one of exon 43, exon 46, exons 50-53 of the DMD pre- mRNA in a patient, preferably in an isolated cell of a patient, the method com-prising providing said cell and/or said patient with a molecule. The invention also relates to said molecule as such.
L'invention a pour objet une méthode dans laquelle on utilise une molécule pour induire et/ou favoriser le saut d'au moins l'un de l'exon 43, l'exon 46, les exons 50 à 53 du pré-ARNm de la DMD chez un patient, de préférence dans une cellule isolée d'un patient, la méthode comprenant la fourniture à ladite cellule et/ou au dit patient d'une molécule. L'invention concerne également ladite molécule en tant que telle.
Aartsma-Rus Annemieke
de Kimpe Josephus Johannes
Platenburg Gerardus Johannes
Van Deutekom Judith Christina Theodora
Van Ommen Garrit-Jan Boudewijn
Academisch Ziekenhuis Leiden
Prosensa B.v.
Prosensa Holding Bv
Prosensa Technologies B.v.
Ridout & Maybee Llp
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