公开(公告)号：US20230057380A1

公开(公告)日：2023-02-23

申请号：US17779793

申请日：2020-11-25

申请人： University of Massachusetts ,  Chengdu Kanghong Biotechnology Co. Ltd.

发明人： Guangping Gao ,  Phillip Tai ,  Claudio Punzo ,  Zunhong Ke

IPC分类号： C12N7/00 ,  C07K16/22 ,  C12N15/86 ,  A61K9/00

摘要： Aspects of the disclosure relate to a recombinant adeno-virus encoding an anti-Vascular endothelial cell growth factor (VEGF) agent in a cell or subject (e.g., rAAV2.7m8-KH902). In some embodiments, compositions described herein are useful for treating subjects having diseases associated with angiogenesis or aberrant VEGF activity/signaling.

公开(公告)号：US11920168B2

公开(公告)日：2024-03-05

申请号：US16775311

申请日：2020-01-29

申请人： University of Massachusetts

发明人： Guangping Gao ,  Phillip D. Zamore ,  Dan Wang

IPC分类号： C12N9/22 ,  A61K38/00 ,  A61K48/00 ,  C07K14/005 ,  C12N7/00 ,  C12N15/11 ,  C12N15/90

CPC分类号： C12N9/22 ,  A61K48/005 ,  C07K14/005 ,  C12N7/00 ,  C12N15/11 ,  C12N15/907 ,  A61K38/00 ,  C07K2319/735 ,  C12N2310/20 ,  C12N2750/14121 ,  C12N2750/14122 ,  C12N2750/14142 ,  C12N2750/14143

摘要： The disclosure in some aspects relates to recombinant adeno-associated viruses having nuclease grafted to one or more capsid proteins. In some aspects, the disclosure relates to isolated AA V capsid proteins having terminally grafted nucleases and isolated nucleic acids encoding the same. Recent approaches to delivering nucleases to cells for gene editing have focused on delivering of expression vectors engineered to express the nucleases in target cells. However, these approaches have proved to be problematic in many instances due to genotoxicity resulting from to prolonged expression of gene editing system in vivo. To prevent such off-target genotoxicity due to prolonged presence of a gene editing system, several studies explored delivery of mRNA or protein instead of delivering the gene coding for the nucleases in cell culture.

公开(公告)号：US20230416779A1

公开(公告)日：2023-12-28

申请号：US18311367

申请日：2023-05-03

申请人： University of Massachusetts

发明人： Miguel Sena Esteves ,  Guangping Gao ,  Michael R. Green ,  Dan Wang ,  Tessa Mercedes Simone

IPC分类号： C12N15/86 ,  C07K14/47

CPC分类号： C12N15/86 ,  C07K14/4702 ,  C12N2750/14143 ,  C12N2310/141 ,  C12N2330/10 ,  C07K2319/41

摘要： In some aspects, the disclosure relates to compositions and methods of engineering a transgene. In some embodiments, the disclosure provides self-regulating recombinant nucleic acids, viral vectors and pharmaceutical compositions comprising a MeCP2 transgene. In some embodiments, compositions and methods described by the disclosure are useful for treating diseases and disorders associated with a loss of function mutation, for example Rett syndrome.

公开(公告)号：US11826434B2

公开(公告)日：2023-11-28

申请号：US17130084

申请日：2020-12-22

申请人： University of Massachusetts

发明人： Guangping Gao ,  Phillip D. Zamore

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

CPC分类号： A61K48/0008 ,  A01K67/0275 ,  A61K9/0019 ,  A61K38/00 ,  A61K48/0058 ,  C12N7/00 ,  C12N15/111 ,  C12N15/113 ,  C12N15/8509 ,  C12N15/86 ,  A01K2207/05 ,  A01K2217/058 ,  A01K2227/105 ,  A01K2267/03 ,  A01K2267/0331 ,  C12N2015/8527 ,  C12N2310/13 ,  C12N2310/141 ,  C12N2320/32 ,  C12N2330/51 ,  C12N2710/10342 ,  C12N2750/14143 ,  C12N2750/14145 ,  C12N2820/002 ,  C12N2840/007 ,  C12N2840/102

摘要： 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：US11739346B2

公开(公告)日：2023-08-29

申请号：US16500238

申请日：2018-04-05

申请人： University of Massachusetts

发明人： Guangping Gao ,  Hemant Khanna

IPC分类号： C07H21/04 ,  C12N15/63 ,  A61K31/70 ,  C12N15/86 ,  A61P27/02 ,  A61K9/00 ,  A61K35/761 ,  C07K14/47 ,  C12N7/00 ,  A01K67/00 ,  A61K48/00

CPC分类号： C12N15/86 ,  A61K9/0048 ,  A61K35/761 ,  A61P27/02 ,  C07K14/47 ,  C12N7/00 ,  A61K48/00 ,  C12N2750/14143

摘要： Aspects of the disclosure relate to compositions and methods useful for treating ocular ciliopathies, for example Leber congenital amaurosis (LCA). In some embodiments, the disclosure provides isolated nucleic acids comprising a transgene encoding a CEP290 protein fragment, and methods of treating ocular ciliopathies using the same.

公开(公告)号：US20230073187A1

公开(公告)日：2023-03-09

申请号：US17813451

申请日：2022-07-19

申请人： University of Massachusetts

发明人： Guangping Gao ,  Jianzhong Ai ,  Hong Li ,  Qiang Wei

IPC分类号： A61K48/00 ,  C12N15/86 ,  C12N5/10 ,  C07K14/075 ,  C07K14/005 ,  A61K9/00 ,  A61K38/17 ,  A61P35/00

摘要： The disclosure relates to compositions and methods for rAAV-mediated delivery of a transgene to a subject. In some embodiments, the rAAV transduces the prostate tissue of a subject. In some embodiments, the methods are useful for treatment of prostate disease (e.g., prostatitis, BPH, prostate cancer).

公开(公告)号：US20210180031A1

公开(公告)日：2021-06-17

申请号：US17116168

申请日：2020-12-09

申请人： University of Massachusetts

发明人： Guangping Gao ,  Li Zhong

IPC分类号： C12N7/00 ,  C12N15/864 ,  A61K38/17 ,  A61K48/00 ,  C12N15/113 ,  C12N15/86

摘要： 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-associate viruses. In some aspects, the disclosure relates to isolated AAV capsid proteins and isolated nucleic acids encoding the same.

公开(公告)号：US10894949B2

公开(公告)日：2021-01-19

申请号：US16058938

申请日：2018-08-08

申请人： University of Massachusetts

发明人： Guangping Gao ,  Li Zhong

IPC分类号： A61K48/00 ,  A61K38/17 ,  C12N15/113 ,  C12N15/86 ,  C12N7/00 ,  C12N15/864

摘要： 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-associate viruses. In some aspects, the disclosure relates to isolated AAV capsid proteins and isolated nucleic acids encoding the same.

公开(公告)号：US10829783B2

公开(公告)日：2020-11-10

申请号：US15367708

申请日：2016-12-02

申请人： University of Massachusetts

发明人： Guangping Gao ,  Jun Xie

IPC分类号： C12N15/86 ,  C12N15/113 ,  C12N7/00

摘要： Some aspects of this invention provide nucleic acid constructs for transgene expression. Some aspects of this invention provide multicistronic nucleic acid constructs, for example, comprising an expression cassette encoding a hairpin RNA and a reporter expression cassette. Some aspects of this invention provide nucleic acid constructs comprising two or more self-complementary nucleic acid sequences, for example, hairpin RNA encoding nucleic acid sequences and AAV inverse terminal repeats. Methods for the use of the constructs in therapy and research are also provided.

公开(公告)号：US10689420B2

公开(公告)日：2020-06-23

申请号：US16019636

申请日：2018-06-27

申请人： University of Massachusetts

发明人： Guangping Gao ,  Terence Flotte ,  Jun Xie

IPC分类号： C12N15/00 ,  C12N7/00 ,  A61K39/23 ,  C07K14/005 ,  C12N15/86 ,  A61K31/7088 ,  A61K35/76 ,  A61K48/00

摘要： 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.