van Ommen, Garrit-Jan Boudewijn,van Deutekom, Judith Christina Theodora,Den Dunnen, Johannes Theodorus
申请号：
ES20150166264T
公开号：
ES2690049(T3)
申请日:
2001.09.21
申请国别(地区):
西班牙
年份:
2018
代理人:
摘要：
The present invention provides a method for at least in part decreasing the production of an aberrant protein in a cell, said cell comprising pre-mRNA comprising exons coding for said protein, by inducing so-called exon skipping in said cell. Exon-skipping results in mature mRNA that does not contain the skipped exon which leads to an altered product if said exon codes for amino acids. Exon skipping is performed by providing a cell with an agent capable of specifically inhibiting an exon inclusion signal, for instance an exon recognition sequence, of said exon. Said exon inclusion signal can be interfered with by a nucleic acid comprising complementarity to a part of said exon. Said nucleic acid, which is also herewith provided, can be used for the preparation of a medicament, for instance for the treatment of an inherited disease.
