Modulation of exon recognition in pre-mRNA by interfering with the secondary RNA structure

a technology of exon recognition and secondary rna, which is applied in the field of molecular biology and medicine, can solve the problems of reducing the risk that also one or more other pre-mrna will be able to hybridize to the oligonucleotide, disrupting the exon inclusion signal, etc., and achieves strong secondary structures. , the effect of improving the invasion efficiency of the oligonucleotide and increasing efficiency
US20060099616A1Inactive Publication Date: 2006-05-11ACADEMISCH ZIEKENHUIS BIJ DE UNIV VAN AMSTERDAM ACADEMISCH MEDISCH CENT
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Patent Information

Authority / Receiving Office
US · United States
Patent Type
Applications(United States)
Current Assignee / Owner
ACADEMISCH ZIEKENHUIS BIJ DE UNIV VAN AMSTERDAM ACADEMISCH MEDISCH CENT
Publication Date
2006-05-11
Estimated Expiration
Not applicable · inactive patent

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Abstract

The invention provides a method for generating an oligonucleotide with which an exon may be skipped in a pre-mRNA and thus excluded from a produced mRNA thereof. Further provided are methods for altering the secondary structure of an mRNA to interfere with splicing processes and uses of the oligonucleotides and methods in the treatment of disease. Further provided are pharmaceutical compositions and methods and means for inducing skipping of several exons in a pre-mRNA.
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Description

CROSS-REFERENCE TO RELATED APPLICATION

[0001] This application is a continuation of PCT International Patent Application No. PCT / NL2004 / 000196 filed on Mar. 22, 2004, designating the United States of America, and published in English, as PCT International Publication No. WO 2004 / 083446 A2 on Sep. 30, 2004, which application claims priority to PCT / NL03 / 00214, filed on Mar. 21, 2003, the contents of the entirety of each of which are incorporated herein by this reference.STATEMENT ACCORDING TO 37 C.F.R. § 1.52(e)(5)—SEQUENCE LISTING SUBMITTED ON COMPACT DISC

[0002] Pursuant to 37 C.F.R. § 1.52(e)(1)(iii), a compact disc containing an electronic version of the Sequence Listing has been submitted concomitant with this application, the contents of which are hereby incorporated by reference. A second compact disc is submitted and is an identical copy of the first compact disc. The discs are labeled “copy 1” and “copy 2,” respectively, and each disc contains one file entitled “sequence list...

Claims

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