公开(公告)号：US20240360475A1

公开(公告)日：2024-10-31

申请号：US18771850

申请日：2024-07-12

申请人： NEW YORK UNIVERSITY

发明人： Dounia ABBADI ,  Robert J. SCHNEIDER

IPC分类号： C12N15/86 ,  A61P21/00

CPC分类号： C12N15/86 ,  A61P21/00 ,  C12N2750/14143

摘要： The present application relates to an adeno-associated viral (AAV) vector, comprising a muscle cell-specific promoter and a nucleic acid molecule encoding an AU-rich mRNA binding factor 1 (AUF1) protein or a functional fragment thereof, where the nucleic acid molecule is heterologous to and operatively coupled to the muscle cell-specific promoter. Also disclosed are compositions comprising the AAV vector, as well as methods of promoting muscle regeneration in injured muscle, a method of treating degenerative skeletal muscle loss in a subject, methods of preventing traumatic muscle injury in a subject such as Duchenne Muscular Dystrophy, methods of treating traumatic muscle injury in a subject, and methods of treating muscle loss due to aging in a subject.

公开(公告)号：US20210222199A1

公开(公告)日：2021-07-22

申请号：US17152463

申请日：2021-01-19

申请人： NEW YORK UNIVERSITY

发明人： Dounia ABBADI ,  Robert J. SCHNEIDER

IPC分类号： C12N15/86 ,  A61P21/00

摘要： The present application relates to an adeno-associated viral (AAV) vector, comprising a muscle cell-specific promoter and a nucleic acid molecule encoding an AU-rich mRNA binding factor 1 (AUF1) protein or a functional fragment thereof, where the nucleic acid molecule is heterologous to and operatively coupled to the muscle cell-specific promoter. Also disclosed are compositions comprising the AAV vector, as well as methods of promoting muscle regeneration in injured muscle, a method of treating degenerative skeletal muscle loss in a subject, methods of preventing traumatic muscle injury in a subject such as Duchenne Muscular Dystrophy, methods of treating traumatic muscle injury in a subject, and methods of treating muscle loss due to aging in a subject.