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US 8,361,979 B2Granted
Means and method for inducing exon-skipping
AssigneeAcademisch Ziekenhuis Leiden
InventorsAnnemieke Aartsma-Rus, Judith Christina T. Van Deutekom, Garrit-Jan Boudewijn Van Ommen
Life of this patent
Estimating the term…
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.
Claims7
An AON comprising a sequence selected from the group consisting of:
- SEQ ID NO:115 to SEQ ID NO:118.
Dependencies are inferred from the claim text.