发明名称 |
Antisense oligonucleotides which combat aberrant splicing and methods of using the same |
摘要 |
A method of combatting aberrant splicing in a pre-mRNA molecule containing a mutation is disclosed. When present in the pre-mRNA, the mutation causes the pre-mRNA to splice incorrectly and produce an aberrant mRNA or mRNA fragment different from the mRNA ordinarily encoded by the pre-mRNA. The method comprises hybridizing an antisense oligonucleotide to the pre-mRNA molecule to create a duplex molecule under conditions which permit splicing. The antisense oligonucleotide is one which does not activate RNase H, and is selected to block a member of the aberrant set of splice elements created by the mutation so that the native intron is removed by splicing and the first mRNA molecule encoding a native protein is produced. Oligonucleotides useful for carrying out the method are also disclosed.
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申请公布号 |
US5916808(A) |
申请公布日期 |
1999.06.29 |
申请号 |
US19970802384 |
申请日期 |
1997.02.19 |
申请人 |
THE UNIVERSITY OF NORTH CAROLINA AT CHAPEL HILL |
发明人 |
KOLE, RYSZARD;DOMINSKI, ZBIGNIEW |
分类号 |
C12N15/09;A61K31/70;A61K38/00;A61K48/00;C07H21/04;C12N5/10;C12N15/113;C12P21/02;(IPC1-7):C12Q1/68;C07H21/00 |
主分类号 |
C12N15/09 |
代理机构 |
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代理人 |
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主权项 |
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地址 |
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