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    GENE THERAPEUTICS FOR TREATING BONE DISORDERS
    1.
    发明申请
    GENE THERAPEUTICS FOR TREATING BONE DISORDERS 有权

    公开(公告)号：US20210023241A1

    公开(公告)日：2021-01-28

    申请号：US16982640

    申请日：2019-03-22

    Applicant: University of Massachusetts

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

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

    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).

    DEVELOPMENT OF NOVEL GENE THERAPEUTICS FOR INFLAMMATION-INDUCED BONE LOSS
    2.
    发明公开
    DEVELOPMENT OF NOVEL GENE THERAPEUTICS FOR INFLAMMATION-INDUCED BONE LOSS 审中-公开

    公开(公告)号：US20240002844A1

    公开(公告)日：2024-01-04

    申请号：US18021901

    申请日：2021-08-18

    Applicant: University of Massachusetts

    Inventor： Jae-Hyuck Shim ,  Guangping Gao ,  Jun Xie ,  Yeon-Suk Yang

    IPC: C12N15/113 ,  C12N15/86 ,  A61P29/00 ,  A61P19/02

    CPC classification number: C12N15/113 ,  C12N15/86 ,  C12N2750/14143 ,  A61P19/02 ,  C12N2310/141 ,  A61P29/00

    Abstract: Aspects of the disclosure relate to compositions and methods for reding inflammation and/or inhibiting bone loss (e.g., bone loss induced by inflammation). In some embodiments, the disclosure provides isolated nucleic acids and expression constructs (e.g., rAAVs, etc.) that encode one or more of the following transgenes: inhibitory nucleic acids targeting Schnurri 3 (SHN3), inhibitory nucleic acids targeting Cathepsin K (CTSK), inhibitory nucleic acids targeting sclerostin (SOST), inhibitory nucleic acids targeting receptor activator of NF-κβ (RANK), inhibitory nucleic acids targeting receptor activator of NF-κβ ligand (RANKL), soluble TNF-α Receptor 2 (sTNFR2), and soluble IL-1 Receptor Antagonist (sIL1Rα). In some embodiments, compositions described by the disclosure are useful for treating diseases associated with inflammation, for example rheumatoid arthritis (RA).

    Gene therapeutics for treating bone disorders
    4.
    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).

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