公开(公告)号：US12221609B2

公开(公告)日：2025-02-11

申请号：US17271982

申请日：2019-09-20

Applicant: University of Connecticut

Inventor： Stormy Chamberlain ,  Noelle Germain

IPC: C12N15/113 ,  C12N15/86

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.

公开(公告)号：US20230332150A1

公开(公告)日：2023-10-19

申请号：US18179716

申请日：2023-03-07

Applicant: University of Connecticut ,  Ovid Therapeutics Inc.

Inventor： Stormy CHAMBERLAIN ,  Noelle Germain ,  Peter Perrino

IPC: C12N15/113 ,  C12N15/86

CPC classification number: C12N15/113 ,  C12N15/86 ,  C12N15/1137 ,  C12N2310/122 ,  C12N2310/531 ,  C12N2740/15043

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.

公开(公告)号：US20230332157A1

公开(公告)日：2023-10-19

申请号：US18179726

申请日：2023-03-07

Applicant: University of Connecticut ,  Ovid Therapeutics Inc.

Inventor： Stormy CHAMBERLAIN ,  Noelle Germain ,  Peter Perrino

IPC: C12N15/113 ,  C12N15/86

CPC classification number: C12N15/1137 ,  C12N15/86 ,  C12N2310/122 ,  C12N2310/531 ,  C12N2740/15043

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 UBE3A-ATS in Angelman syndrome using viral vector delivery of short hairpin RNAs.