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    AAV CAPSIDS VARIANTS AND USES THEREOF
    2.
    发明申请
    AAV CAPSIDS VARIANTS AND USES THEREOF 有权

    公开(公告)号：US20230138766A1

    公开(公告)日：2023-05-04

    申请号：US17915548

    申请日：2021-03-30

    申请人： University of Massachusetts ,  Sichuan University

    发明人： Guangping Gao ,  Guangchao Xu ,  Phillip Tai ,  Yuquan Wei ,  Li Luo

    IPC分类号： C12N15/86 ,  A61K9/00 ,  C07K14/005

    摘要： Aspects of the disclosure relate to compositions and methods for delivering a transgene (e.g., a transgene encoding one or more gene products) to a target cell. The disclosure is based, in part, on adeno-associated virus (AAV) capsid protein variants characterized by tropisms for certain cell types (e.g., neurons, muscle cells, bone cells, heart cells, etc.). In some embodiments, recombinant A A Vs (rAAVs) comprising the capsid protein variants (e.g., AAVv66, SEQ ID NO: 1) are more efficiently packaged than rAAVs having certain wild-type AAV capsid proteins. Methods of delivering an rAAV comprising the AAV capsid protein variants are also described by the disclosure.

    CARDIAC CELL REPROGRAMMING WITH MICRORNAS AND OTHER FACTORS
    3.
    发明申请
    CARDIAC CELL REPROGRAMMING WITH MICRORNAS AND OTHER FACTORS 有权

    公开(公告)号：US20230137971A1

    公开(公告)日：2023-05-04

    申请号：US17625376

    申请日：2020-07-10

    申请人： Tenaya Therapeutics Inc.

    发明人： Huanyu ZHOU ,  Chetan SRINATH ,  Timothy HOEY

    IPC分类号： A61K48/00 ,  C12N15/86 ,  C12N15/113 ,  C07K14/47 ,  C12N5/077 ,  A61P9/10

    摘要： The present disclosure provides methods for generating induced cardiomyocytes by expression of selected microRNAs with MYOCD and ASCL1, or with MYOCD alone. Illustrative microRNAs include miR-133, miR-1, miR-19, and/or miR-20b. The present disclosure further provides gene-delivery vectors comprising one or more polynucleotides encoding a selected microRNA with YOCD, withMYOCD and ASCL1, with MYOCD-2A-ASCL1, or with ASCL1-2A-MYOCD. It further provides methods of using such compositions and vectors, or induced cardiomyocytes generated with these factors, for treating a heart condition.

    AD7-VECTORED VACCINE FOR PREVENTING SARS- COV-2 INFECTION
    9.
    发明申请
    AD7-VECTORED VACCINE FOR PREVENTING SARS- COV-2 INFECTION 有权

    公开(公告)号：US20230127808A1

    公开(公告)日：2023-04-27

    申请号：US17598659

    申请日：2021-02-02

    申请人： GUANGZHOU N BIOMED LTD.

    发明人： Ling Chen ,  Suhua Guan ,  Chenchen Yang ,  Qian Wang

    IPC分类号： A61K39/215 ,  C12N15/86 ,  A61P31/14 ,  A61P11/00 ,  A61K45/06

    摘要： Disclosed is an Ad7-vectored vaccine for preventing SARS-CoV-2 infection, which is an adenovirus vectored vaccine, comprising an Ad7 vector loaded with a nucleic acid sequence shown in SEQ ID NO: 1. Some embodiments of the disclosure have good safety and are convenient to use. Experiments have shown that the vaccine is capable of producing more S protein in human cells, which is expected to be developed as a vaccine for preventing SARS-CoV-2 infection. Some embodiments of the disclosure may be used in combination with other vaccines, and may also be used as therapeutic vaccines for COVID-19. When a patient is vaccinated with the Ad7-vectored vaccine of the present disclosure at the initial stage of infection, the vaccine can quickly induces immune response in the human body, thereby achieving a therapeutic effect.