公开(公告)号：US09701984B2

公开(公告)日：2017-07-11

申请号：US14445670

申请日：2014-07-29

Applicant: University of Massachusetts

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

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

CPC classification number: C12N15/86 ,  A61K31/713 ,  A61K38/50 ,  A61K48/00 ,  A61K48/0058 ,  A61K48/0075 ,  C12N7/00 ,  C12N9/80 ,  C12N15/1137 ,  C12N15/635 ,  C12N15/8645 ,  C12N2310/141 ,  C12N2750/14133 ,  C12N2750/14141 ,  C12N2750/14143 ,  C12N2750/14145 ,  C12N2750/14162 ,  C12N2810/10 ,  C12N2840/007 ,  C12Y305/01015

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：US20160222067A1

公开(公告)日：2016-08-04

申请号：US15007559

申请日：2016-01-27

Applicant: University of Massachusetts

Inventor： Guangping Gao ,  Terence Flotte ,  Jun Xie

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

CPC classification number: C07K14/005 ,  A61K31/7088 ,  A61K35/76 ,  A61K48/00 ,  C12N7/00 ,  C12N15/86 ,  C12N2750/14122 ,  C12N2750/14143 ,  C12N2750/14145

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.

Abstract translation: 本发明在某些方面涉及具有不同组织靶向能力的重组腺相关病毒。 在一些方面，本发明涉及使用重组腺相关病毒的基因转移方法。 在一些方面，本发明涉及分离的AAV衣壳蛋白和编码其的分离的核酸。

公开(公告)号：US20160135438A1

公开(公告)日：2016-05-19

申请号：US14940574

申请日：2015-11-13

Applicant: University of Massachusetts

Inventor： Guangping Gao ,  Phillip D. Zamore

IPC: A01K67/027 ,  C12N15/86 ,  C12N15/85

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

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.

Abstract translation: 本发明在一些方面涉及用于鉴定细胞中腺相关病毒的分离的核酸，组合物和试剂盒。 在一些方面，本发明提供用于使用重组AAV（rAAV）产生体细胞转基因动物模型的试剂盒和方法，所述重组AAV（rAAV）具有至少一个表达小干扰核酸或至少一个miRNA结合位点的转基因。

公开(公告)号：US20240409940A1

公开(公告)日：2024-12-12

申请号：US18746105

申请日：2024-06-18

Applicant: University of Massachusetts

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

IPC: C12N15/113 ,  A61K31/713 ,  C12N15/11 ,  C12N15/86

Abstract: The present disclosure relates to the field of rAAV delivery of transgenes. In some aspects, the disclosure relates to RNAi. Provided herein are recombinant adeno-associated virus (rAAV) vectors comprising modified ITRs. In some embodiments, the modified ITRs comprise a sequence encoding a shRNA, miRNA, or AmiRNA.

公开(公告)号：US11920168B2

公开(公告)日：2024-03-05

申请号：US16775311

申请日：2020-01-29

Applicant: University of Massachusetts

Inventor： Guangping Gao ,  Phillip D. Zamore ,  Dan Wang

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

CPC classification number: 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

Abstract: 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

Applicant: University of Massachusetts

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

IPC: C12N15/86 ,  C07K14/47

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

Abstract: 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

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

CPC classification number: 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

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：US11739346B2

公开(公告)日：2023-08-29

申请号：US16500238

申请日：2018-04-05

Applicant: University of Massachusetts

Inventor： Guangping Gao ,  Hemant Khanna

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

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

Abstract: 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

Applicant: University of Massachusetts

Inventor： Guangping Gao ,  Jianzhong Ai ,  Hong Li ,  Qiang Wei

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

Abstract: 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

Applicant: University of Massachusetts

Inventor： Guangping Gao ,  Li Zhong

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

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