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US 12,221,609 B2Granted
Compositions and methods to restore paternal UBE3A gene expression in human Angelman syndrome
AssigneeUniversity of Connecticut
InventorsStormy Chamberlain, Noelle Germain
Life of this patent
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Abstract
The present invention relates to compositions and methods for activating expression from the paternally-inherited allele of UBE3A in human Angelman's Syndrome neurons using viral vector delivery of short hairpin RNAs, ribozymes, and/or microRNAs.
Claims28
A polynucleotide comprising a nucleotide sequence encoding a short hairpin RNA (shRNA), wherein the shRNA is capable of inhibiting the silencing of paternal UBE3A gene, and wherein the nucleotide sequence encoding the shRNA consists of SEQ ID NO:
- 2.
Dependencies are inferred from the claim text.