公开(公告)号：US11674154B2

公开(公告)日：2023-06-13

申请号：US17541141

申请日：2021-12-02

Applicant: University of Massachusetts

Inventor： Jae-Hyuck Shim ,  Guangping Gao ,  Jun Xie ,  Yeon-Suk Yang ,  Jung min Kim ,  Sachin Chaugule

IPC: C12N15/86 ,  A61P19/08 ,  A61K38/45 ,  A61K48/00 ,  C12N9/22 ,  C12N15/11

CPC classification number: C12N15/86 ,  A61K38/45 ,  A61K48/0066 ,  A61P19/08 ,  C12N9/22 ,  C12N15/111 ,  C12Y207/1103 ,  C12N2310/141 ,  C12N2310/20 ,  C12N2320/34 ,  C12N2750/14143 ,  C12N2830/008

Abstract: In some aspects, the disclosure relates to compositions and methods for treating fibrodysplasia ossificans progressiva (FOP) in a subject. In some aspects, the disclosure provides isolated nucleic acids, and vectors such as rAAV vectors, configured to express transgenes that inhibit (e.g., decrease) expression of mutated AVCR1 gene in muscle cells or connective tissues.

公开(公告)号：US20230295662A1

公开(公告)日：2023-09-21

申请号：US18304472

申请日：2023-04-21

Applicant: University of Massachusetts

Inventor： Jae-Hyuck Shim ,  Guangping Gao ,  Jun Xie ,  Yeon-Suk Yang ,  Jung min Kim ,  Sachin Chaugule

IPC: C12N15/86 ,  A61K38/45 ,  A61K48/00 ,  A61P19/08 ,  C12N9/22 ,  C12N15/11

CPC classification number: C12N15/86 ,  A61K38/45 ,  A61K48/0066 ,  A61P19/08 ,  C12N9/22 ,  C12Y207/1103 ,  C12N15/111 ,  C12N2830/008 ,  C12N2310/141 ,  C12N2310/20 ,  C12N2320/34 ,  C12N2750/14143

Abstract: In some aspects, the disclosure relates to compositions and methods for treating fibrodysplasia ossificans progressiva (FOP) in a subject. In some aspects, the disclosure provides isolated nucleic acids, and vectors such as rAAV vectors, configured to express transgenes that inhibit (e.g., decrease) expression of mutated AVCR1 gene in muscle cells or connective tissues.

公开(公告)号：US20220177915A1

公开(公告)日：2022-06-09

申请号：US17541141

申请日：2021-12-02

Applicant: University of Massachusetts

Inventor： Jae-Hyuck Shim ,  Guangping Gao ,  Jun Xie ,  Yeon-Suk Yang ,  Jung Min Kim ,  Sachin Chaugule

IPC: C12N15/86 ,  C12N9/22 ,  A61P19/08 ,  C12N15/11

Abstract: In some aspects, the disclosure relates to compositions and methods for treating fibrodysplasia ossificans progressiva (FOP) in a subject. In some aspects, the disclosure provides isolated nucleic acids, and vectors such as rAAV vectors, configured to express transgenes that inhibit (e.g., decrease) expression of mutated AVCR1 gene in muscle cells or connective tissues.