发明公开
EP2389390A2 MEANS AND METHODS FOR MODULATING NOTCH3 PROTEIN EXPRESSION AND/OR THE CODING REGION OF NOTCH3; COMPOSITIONS AND USE THEREOF IN THE TREATMENT OF CADASIL 审中-公开
手段和一种用于调节NOTCH3蛋白的表达和/或编码NOTCH3,组合物及其使用区域CADASIL的治疗

MEANS AND METHODS FOR MODULATING NOTCH3 PROTEIN EXPRESSION AND/OR THE CODING REGION OF NOTCH3; COMPOSITIONS AND USE THEREOF IN THE TREATMENT OF CADASIL
摘要:
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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