Academisch Ziekenhuis Leiden (h.o.d.n. Lumc)

Biomarin Technologies B.v.

Academisch Ziekenhuis Leiden

Academisch Ziekenbuls Leiden

Adriana Marie Rus

Garrit-Jan Boudewijn Van Ommen

Judith Christina Theodora Van Deutekom

Gerard Johannes Platenburg

Josephus Johannes De Kimpe

Saskia Anne-Marie Jenny Lesnik Oberstein

Johannes Gregorius Dauwerse

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.

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

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 the exon. The invention further relates to such molecule used in the method.

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

The invention among other provides means and methods for modulating NOTCH3 expression and/or protein coding domain. In one aspect the invention provides a method for at least reducing an elevated level of NOTCH3 protein in a NOTCH3 expressing cell or the immediate vicinity thereof said method comprising providing said cell with an anti-sense oligonucleotide specific for NOTCH3 mRNA or pre-mRNA thereby decreasing production of said NOTCH3 protein or thereby altering the protein coding region in said NOTCH3 mRNA or pre-mRNA.

Means and methods for modulating NOTCH3 protein expression and/or the coding region of NOTCH3; compositions and use thereof in the treatment of CADASIL

The invention among other provides means and methods for modulating NOTCH3 expression and/or protein coding domain. In one aspect the invention provides a method for at least reducing an elevated level of NOTCH3 protein in a NOTCH3 expressing cell or the immediate vicinity thereof said method comprising providing said cell with an anti-sense oligonucleotide specific for NOTCH3 m RNA or pre-m RNA thereby decreasing production of said NOTCH3 protein or thereby altering the protein coding region in said NOTCH3 m RNA or pre-m RNA.

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Hoofddorp, Netherlands

Adriana Marie Rus