公开(公告)号：US20210317475A1

公开(公告)日：2021-10-14

申请号：US17193030

申请日：2021-03-05

Applicant: University of Massachusetts

Inventor： Guangping Gao ,  Phillip D. Zamore ,  Shaoyong Li

IPC: C12N15/86 ,  C12N15/113 ,  A61K38/17 ,  A61K38/28 ,  A61K38/44 ,  A61K38/48 ,  A61K38/51 ,  A61K48/00 ,  C07K16/10 ,  C07K16/22 ,  C07K16/28 ,  C07K16/32 ,  C12N7/00 ,  C12N15/117

Abstract: The disclosure in some aspects, relates to nucleic acids, compositions and kits useful for gene therapy with reduced immune response to transgene products.

公开(公告)号：US20210205476A1

公开(公告)日：2021-07-08

申请号：US17130084

申请日：2020-12-22

Applicant: University of Massachusetts

Inventor： Guangping Gao ,  Phillip D. Zamore

IPC: A61K48/00 ,  C12N15/11 ,  C12N15/86 ,  C12N15/85 ,  A01K67/027 ,  A61K38/00 ,  C12N7/00 ,  C12N15/113 ,  A61K9/00

Abstract: The invention in some aspects relates to isolated nucleic acids, compositions, and kits useful for identifying adeno-associated viruses in cells. In some aspects, the invention provides kits and methods for producing somatic transgenic animal models using recombinant AAV (rAAV) to an animal having at least one transgene that expresses a small interfering nucleic acid or at least one binding site for a miRNA.

公开(公告)号：US10975391B2

公开(公告)日：2021-04-13

申请号：US15306543

申请日：2015-04-24

Applicant: University of Massachusetts

Inventor： Guangping Gao ,  Phillip D. Zamore ,  Shaoyong Li

IPC: C12N15/86 ,  C12N15/113 ,  A61K31/713 ,  C12N7/00 ,  C12N15/117 ,  A61K38/17 ,  A61K38/28 ,  A61K38/44 ,  A61K38/48 ,  A61K38/51 ,  A61K48/00 ,  C07K16/10 ,  C07K16/22 ,  C07K16/28 ,  C07K16/32

Abstract: The disclosure in some aspects, relates to nucleic acids, compositions and kits useful for gene therapy with reduced immune response to transgene products.

公开(公告)号：US10905776B2

公开(公告)日：2021-02-02

申请号：US16373718

申请日：2019-04-03

Applicant: University of Massachusetts

Inventor： Guangping Gao ,  Phillip D. Zamore

IPC: C12N15/86 ,  A61K48/00 ,  C12N15/11 ,  C12N15/85 ,  A01K67/027 ,  A61K38/00 ,  C12N7/00 ,  C12N15/113 ,  A61K9/00

Abstract: The invention in some aspects relates to isolated nucleic acids, compositions, and kits useful for identifying adeno-associated viruses in cells. In some aspects, the invention provides kits and methods for producing somatic transgenic animal models using recombinant AAV (rAAV) to an animal having at least one transgene that expresses a small interfering nucleic acid or at least one binding site for a miRNA.

公开(公告)号：US20200377554A1

公开(公告)日：2020-12-03

申请号：US16870816

申请日：2020-05-08

Applicant: University of Massachusetts

Inventor： Guangping Gao ,  Terence Flotte ,  Jun Xie

IPC: C07K14/005 ,  C12N15/86 ,  A61K31/7088 ,  A61K35/76 ,  C12N7/00

Abstract: The invention in some aspects relates to recombinant adeno-associated viruses having distinct tissue targeting capabilities. In some aspects, the invention relates to gene transfer methods using the recombinant adeno-associate viruses. In some aspects, the invention relates to isolated AAV capsid proteins and isolated nucleic acids encoding the same.

公开(公告)号：US10731178B2

公开(公告)日：2020-08-04

申请号：US15613646

申请日：2017-06-05

Applicant: University of Massachusetts

Inventor： Guangping Gao ,  Hongwei Zhang ,  Hongyan Wang ,  Zuoshang Xu

IPC: C12N15/86 ,  A61K48/00 ,  C12N15/113 ,  C12N15/864 ,  A61K38/50 ,  A61K31/713 ,  C12N9/80 ,  A61P25/00 ,  C12N7/00 ,  C12N15/63

Abstract: The invention in some aspects relates to recombinant adeno-associated viruses useful for targeting transgenes to CNS tissue, and compositions comprising the same, and methods of use thereof. In some aspects, the invention provides methods and compositions for treating CNS-related disorders.

公开(公告)号：US10480011B2

公开(公告)日：2019-11-19

申请号：US15520977

申请日：2015-10-21

Applicant: University of Massachusetts

Inventor： Guangping Gao ,  Jun Xie ,  Terence Flotte

IPC: C12N15/86 ,  C07K14/005 ,  A01K67/027 ,  A61K48/00 ,  C12N7/00 ,  C12N15/113 ,  C12N15/85 ,  A61K35/00

Abstract: The disclosure in some aspects relates to recombinant adeno-associated viruses having distinct tissue targeting capabilities. In some aspects, the disclosure relates to gene transfer methods using the recombinant adeno-associated viruses. In some aspects, the disclosure relates to isolated AAV capsid proteins and isolated nucleic acids encoding the same.

公开(公告)号：US20190282709A1

公开(公告)日：2019-09-19

申请号：US16373718

申请日：2019-04-03

Applicant: University of Massachusetts

Inventor： Guangping Gao ,  Phillip D. Zamore

IPC: A61K48/00 ,  A61K38/00 ,  C12N15/113 ,  C12N7/00 ,  A01K67/027 ,  C12N15/86 ,  C12N15/11 ,  C12N15/85 ,  A61K9/00

Abstract: The invention in some aspects relates to isolated nucleic acids, compositions, and kits useful for identifying adeno-associated viruses in cells. In some aspects, the invention provides kits and methods for producing somatic transgenic animal models using recombinant AAV (rAAV) to an animal having at least one transgene that expresses a small interfering nucleic acid or at least one binding site for a miRNA.

公开(公告)号：US20190125899A1

公开(公告)日：2019-05-02

申请号：US16093813

申请日：2017-04-14

Applicant: University of Massachusetts

Inventor： Guangping Gao ,  Dominic Gessler

IPC: A61K48/00 ,  A61K35/761 ,  A61K38/50 ,  A61K38/45 ,  A61K45/06

Abstract: In some aspects, the disclosure relates to compositions and methods useful for the diagnosis and treatment of diseases associated with a metabolic imbalance in a subject (e.g.,cancer). In some embodiments, the methods comprise administering to a subject an N-acetylaspartate (NAA)-depleting agent or an N-acetylaspartate (NAA)-increasing agent based upon the subject's metabolic profile.

公开(公告)号：US10202600B2

公开(公告)日：2019-02-12

申请号：US14995699

申请日：2016-01-14

Applicant: University of Massachusetts

Inventor： Guangping Gao ,  Phillip D. Zamore ,  Jun Xie

IPC: C12N15/113 ,  A61K48/00 ,  C12Q1/68 ,  C12N15/86 ,  C12N7/00 ,  C12Q1/6897

Abstract: The invention in some aspects relates to methods and compositions for assessing the effectiveness of miRNA inhibitors. In other aspects of the invention, methods and compositions for treating cholesterol related disorders are provided. In one aspect of the invention, miRNA inhibitors against miR-122 and rAAV-based compositions comprising the same are provided.