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

Johannes Gregorius Dauwerse

Garrit-Jan Boudewijn Van Ommen

Adriana Marie Rus

Saskia Anne-Marie Jenny Lesnik Oberstein

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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Delft, Netherlands

Johannes Gregorius Dauwerse