Modulation of ube3a-ats expression
AssigneeIonis Pharmaceuticals, Inc.
InventorsAmanda Ward, Frank Rigo
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Abstract
Certain embodiments are directed to methods and compounds for inhibiting UBE3A-ATS, the endogenous antisense transcript of ubiquitin protein ligase E3A (UBE3A). Such methods and compounds are useful for. Several embodiments provided herein relate to the discovery that antisense compounds targeting UBE3A-ATS induce paternal expression of UBE3A. Several embodiments are drawn to methods and compounds for inducing paternal expression of UBE3A using antisense compounds targeting UBE3A-ATS within a region of UBE3A-ATS that includes the sequence of the small nucleolar RNA (snoRNA), HBII-85 (also referred to as SNORD116).
Claims134
A method of inducing expression of paternal UBE3A in a cell comprising contacting the cell with an antisense compound targeted to a region of UBE3A-ATS that contains the pre-mRNA for SNORD116.
A method of inducing expression of paternal UBE3A in an animal comprising administering to the animal an antisense compound targeted to UBE3A-ATS that contains the pre-mRNA for SNORD116.
The method of claim 24, wherein UBE3A-ATS comprises a nucleic acid sequence at least 85% identical to SEQ ID NO:
- 1.
A method of treating an animal with an antisense compound targeted to UBE3A-ATS comprising selecting an animal in need thereof and administering to the animal an antisense compound targeted to UBE3A-ATS that contains the pre-mRNA for SNORD116.
A compound comprising an antisense compound targeted to a region of UBE3A-ATS that contains the pre-mRNA for SNORD116.
A method comprising administering to a cell or animal an antisense compound targeted to UBE3A-ATS that contains the pre-mRNA for SNORD116, wherein expression of paternal UBE3A is induced.
Dependencies are inferred from the claim text.