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    NOVEL AAV'S AND USES THEREOF
    65.
    发明申请
    NOVEL AAV'S AND USES THEREOF 有权
    新款AAV及其用途

    公开(公告)号：US20140296486A1

    公开(公告)日：2014-10-02

    申请号：US14246560

    申请日：2014-04-07

    Applicant: University of Massachusetts

    Inventor： Guangping Gao ,  Terence Flotte ,  Jun Xie

    IPC: C07K14/005

    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。

    Gene therapeutics for treating bone disorders
    66.
    发明授权
    Gene therapeutics for treating bone disorders 有权

    公开(公告)号：US12194108B2

    公开(公告)日：2025-01-14

    申请号：US16982640

    申请日：2019-03-22

    Applicant: University of Massachusetts

    Inventor： Jae-Hyuck Shim ,  Guangping Gao ,  Jun Xie ,  Jung Min Kim ,  Dan Wang ,  Yeon-Suk Yang

    IPC: A61K48/00 ,  A61P19/10 ,  C07K14/005 ,  C07K14/635 ,  C12N15/113 ,  C12N15/86

    Abstract: In some aspects, the disclosure relates to compositions and methods for modulating (e.g., increasing and/or decreasing) bone mass in a subject. In some aspects, the disclosure provides isolated nucleic acids, and vectors such as rAAV vectors, configured to express transgenes that promote (e.g., increase) or inhibit (e.g., decrease) activity, differentiation, or function of certain types of bone cells, for example osteoblasts, osteoclasts, osteocytes, etc. In some embodiments, the isolated nucleic acids and vectors described by the disclosure are useful for treating disorders and conditions associated with increased bone mass (e.g., osteopetrosis) or decreased bone mass (e.g., osteoporosis).

    GENE THERAPY FOR SPINAL MUSCULAR ATROPHY
    67.
    发明申请
    GENE THERAPY FOR SPINAL MUSCULAR ATROPHY 有权

    公开(公告)号：US20240424144A1

    公开(公告)日：2024-12-26

    申请号：US18827471

    申请日：2024-09-06

    Applicant: University of Massachusetts

    Inventor： Jun Xie ,  Guangping Gao ,  Qing Xie ,  Hong Ma

    IPC: A61K48/00 ,  A61P21/00 ,  C12N15/86

    Abstract: Aspects of the disclosure relate to compositions and methods for treating spinal muscular atrophy (SMA). The disclosure is based, in part, on isolated nucleic acids and vectors (e.g., viral vectors, such as rAAV vectors) encoding SMN1. In some embodiments, the expression of SMN1 is driven by a native SMN1 promoter or a variant thereof. In some embodiments, isolated nucleic acids and vectors of the disclosure have reduced toxicity and/or increased transgene expression relative to previously described SMN-encoding vectors.

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