Invention Publication
- Patent Title: shRNA TARGETING SNORD115 LOCATIONS TO RESTORE PATERNAL UBE3A GENE EXPRESSION IN ANGELMAN SYNDROME
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Application No.: US18179716Application Date: 2023-03-07
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Publication No.: US20230332150A1Publication Date: 2023-10-19
- Inventor: Stormy CHAMBERLAIN , Noelle Germain , Peter Perrino
- Applicant: University of Connecticut , Ovid Therapeutics Inc.
- Applicant Address: US CT Farmington
- Assignee: University of Connecticut,Ovid Therapeutics Inc.
- Current Assignee: University of Connecticut,Ovid Therapeutics Inc.
- Current Assignee Address: US CT Farmington
- Main IPC: C12N15/113
- IPC: C12N15/113 ; C12N15/86
Abstract:
Provided herein are compositions and methods for activating expression from the paternally-inherited allele of UBE3A in Angelman syndrome using viral vector delivery of short hairpin RNAs. Provided herein are compositions and methods for reducing or eliminating expression of SNORD115 and UBE3A-ATS in Angelman syndrome using viral vector delivery of short hairpin RNAs.
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