Means And Method For Inducing Exon-skipping

Abstract

In the present invention means and method are provided for optimising exon-skipping using exon-internal AON. We show that skipping efficiencies are improved by targeting putative splicing regulatory sequences (ESEs) within an exon. Such double targeting may be particularly useful for exons with which efficient skipping was difficult to obtain prior to the invention.


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Document History
  • Publication: Jan 29, 2013
  • Application: Mar 18, 2007
    US US 30062907 A
  • Priority: May 18, 2007
    EP EP 2007054842 W
  • Priority: May 19, 2006
    EP EP 06076078 A

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