Invention Grant
- Patent Title: Induction of exon skipping in eukaryotic cells
- Patent Title (中): 诱导真核细胞中外显子跳跃
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Application No.: US10395031Application Date: 2003-03-21
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Publication No.: US07973015B2Publication Date: 2011-07-05
- Inventor: Garrit-Jan B. van Ommen , Judith C. T. van Deutekom , Johannes T. den Dunnen
- Applicant: Garrit-Jan B. van Ommen , Judith C. T. van Deutekom , Johannes T. den Dunnen
- Applicant Address: NL Leiden
- Assignee: Academisch Ziekenhuis Leiden
- Current Assignee: Academisch Ziekenhuis Leiden
- Current Assignee Address: NL Leiden
- Agency: Edwards Angell Palmer & Dodge LLP
- Agent Elizabeth Spar; Kathleen Williams
- Priority: EP00203283 20000921
- Main IPC: A61K48/00
- IPC: A61K48/00 ; C07H21/04

Abstract:
The present invention provides a method for at least in part decreasing the production of an aberrant protein in a cell, the cell comprising pre-mRNA comprising exons coding for the protein, by inducing so-called exon skipping in the cell. Exon-skipping results in mature MRNA that does not contain the skipped exon, which leads to an altered product of the 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 the exon. The exon inclusion signal can be interfered with by a nucleic acid comprising complementarity to a part of the exon. The 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.
Public/Granted literature
- US20030235845A1 Induction of exon skipping in eukaryotic cells Public/Granted day:2003-12-25
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