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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.
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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
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.
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公开(公告)号：US11841492B2
公开(公告)日：2023-12-12
申请号：US17274625
申请日：2019-09-13
Applicant: University of Massachusetts
Inventor： David Grunwald , Maximiliaan Huisman , Karina Jouravleva , Carlas Smith , Phillip D. Zamore
Abstract: The invention is directed to a heated stage assembly that can achieve high temperatures (i.e., 40 degrees Celsius and higher and more specifically, 55 degrees Celsius and higher) for use in microscopy including fluorescence microscopy. The microscope stage heater assembly includes a mounting adapter element, a sample holder element mounted within the mounting adapter element, one or more heating elements attached to the sample holder element, and an optical window holder element disposed on a surface of the sample holder element that encloses an optical window element. The optical window element may be in contact with a microscope sample such that the optical window element is heated by the sample holder element to uniformly distribute heat to the specimen.
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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.
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公开(公告)号：US10604754B2
公开(公告)日：2020-03-31
申请号：US14294817
申请日：2014-06-03
Applicant: UNIVERSITY OF MASSACHUSETTS
Inventor： Phillip D. Zamore , Gyorgy Hutvagner , Dianne Schwarz , Martin Simard
IPC: C12N15/113 , C12N15/11 , A61K38/00 , A61K48/00
Abstract: The present invention provides methods of enhancing the efficacy and specificity of RNA silencing. The invention also provides compositions for mediating RNA silencing. In particular, the invention provides siRNAs, siRNA-like molecules, shRNAs, vectors and transgenes having improved specificity and efficacy in mediating silencing of a target gene. Therapeutic methods are also featured.
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Patent Agency Ranking
公开(公告)号：US10385339B2
公开(公告)日：2019-08-20
申请号：US15834383
申请日：2017-12-07
Applicant: UNIVERSITY OF MASSACHUSETTS
Inventor： Phillip D. Zamore , Dianne Schwarz
IPC: C12N15/11 , C12N15/113
Abstract: The present invention provides methods of enhancing the efficacy and specificity of RNAi using single or double blunt-ended siRNA. The invention also provides single and double-blunt ended siRNA compositions, vectors, and transgenes containing the same for mediating silencing of a target gene. Therapeutic methods are also featured.
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公开(公告)号：US09885057B2
公开(公告)日：2018-02-06
申请号：US14952217
申请日：2015-11-25
Applicant: University of Massachusetts
Inventor： Terence Flotte , Christian Mueller , Phillip D. Zamore
CPC classification number: C12N15/86 , A61K31/713 , A61K35/12 , A61K38/57 , A61K48/005 , A61K48/0058 , C07K14/8125 , C12N7/00 , C12N15/111 , C12N15/113 , C12N15/67 , C12N2310/141 , C12N2310/3519 , C12N2320/31 , C12N2750/14121 , C12N2750/14132 , C12N2750/14143
Abstract: The invention relates to isolated nucleic acids and rAAV-based compositions, methods and kits useful for treating genetic diseases (e.g., alpha-1 antitrypsin deficiency).
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公开(公告)号：US20160319279A1
公开(公告)日：2016-11-03
申请号：US15094320
申请日：2016-04-08
Applicant: University of Massachusetts
Inventor： Gyorgy Hutvagner , Phillip D. Zamore
IPC: C12N15/113 , C12Q1/68
CPC classification number: C12N15/113 , C12N15/111 , C12N2310/11 , C12N2310/113 , C12N2310/321 , C12N2320/50 , C12Q1/6897 , C12N2310/3521
Abstract: The present invention relates to the discovery of a method for inhibiting RNA silencing in a target sequence-specific manner. RNA silencing requires a set of conserved cellular factors to suppress expression of gene-encoded polypeptide. The invention provides compositions for sequence-specific inactivation of the RISC component of the RNA silencing pathway, and methods of use thereof. The RISC inactivators of the present invention enable a variety of methods for identifying and characterizing miRNAs and siRNAs, RISC-associated factors, and agents capable of modulating RNA silencing. Therapeutic methods and compositions incorporating RISC inactivators and therapeutic agents identified through use of RISC inactivators are also featured.
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公开(公告)号：US20240240180A1
公开(公告)日：2024-07-18
申请号：US18426444
申请日：2024-01-30
Applicant: University of Massachusetts
Inventor： Terence Flotte , Christian Mueller , Phillip D. Zamore
IPC: C12N15/113 , A61K31/713 , A61K35/12 , A61K38/57 , A61K48/00 , C07K14/81 , C12N7/00 , C12N15/67 , C12N15/86 , C12N15/11
CPC classification number: C12N15/113 , A61K31/713 , A61K35/12 , A61K38/57 , A61K48/005 , A61K48/0058 , C07K14/8125 , C12N7/00 , C12N15/67 , C12N15/86 , C12N15/111 , C12N2310/141 , C12N2310/3519 , C12N2320/31 , C12N2750/14121 , C12N2750/14132 , C12N2750/14143
Abstract: The invention relates to isolated nucleic acids and rAAV-based compositions, methods and kits useful for treating genetic diseases (e.g., alpha-1 antitrypsin deficiency).
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公开(公告)号：US11299734B2
公开(公告)日：2022-04-12
申请号：US16415826
申请日：2019-05-17
Applicant: University of Massachusetts
Inventor： Neil Aronin , Phillip D. Zamore
IPC: C12N15/113 , C12N15/11 , A61K48/00 , C07H21/02 , C12N5/07
Abstract: The present invention relates to the discovery of an effective treatment for a variety of gain-of-function diseases, in particular, Huntington's disease (HD). The present invention utilizes RNA Interference technology (RNAi) against polymorphic regions in the genes encoding various gain-of-function mutant proteins resulting in an effective treatment for the gain-of-function disease.
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