中科微点-全球专利检索

  1. Login
  2. Sign Up

Search Results

131 records (took 0.021 seconds)

    AAV-CAS13D VECTORS AND USES THEREOF
    114.
    发明申请
    AAV-CAS13D VECTORS AND USES THEREOF 有权

    公开(公告)号：US20220186257A1

    公开(公告)日：2022-06-16

    申请号：US17602313

    申请日：2020-04-10

    Applicant: University of Massachusetts

    Inventor： Guangping Gao ,  Alexander Brown

    IPC: C12N15/86 ,  A61P1/16 ,  C12N15/113 ,  C12N15/90 ,  C12N9/22 ,  C12N9/64

    Abstract: Aspects of the disclosure relate to compositions and methods for multiplexed gene silencing in a cell or subject. In some embodiments, the disclosure provides an isolated nucleic acid or an rAAV encoding a transgene comprising a RNA-guided nuclease (RGN) operably linked to a first promoter, and a second promoter operably linked to a multi guide-RNA (multi-gRNA) expression cassette encoding one or more gRNAs targeting a gene associated with hypercholesterolemia or dyslipidemia. In some embodiments, the disclosure provides methods of treating a subject having hypercholesterolemia or dyslipidemia by administering the compositions.

    AAV-MEDIATED GENE THERAPY FOR MAPLE SYRUP URINE DISEASE (MSUD)
    115.
    发明申请
    AAV-MEDIATED GENE THERAPY FOR MAPLE SYRUP URINE DISEASE (MSUD) 有权

    公开(公告)号：US20220162570A1

    公开(公告)日：2022-05-26

    申请号：US17602353

    申请日：2020-04-10

    Applicant: University of Massachusetts ,  Clinic for Special Children

    Inventor： Guangping Gao ,  Dan Wang ,  Jiaming Wang ,  kevin A Strauss

    IPC: C12N9/02 ,  C12N15/86 ,  A61P3/00 ,  A61K48/00

    Abstract: In some aspects the disclosure provides compositions and methods for promoting expression of functional BCKDHA protein, which is the E1-alpha subunit of the branched-chain alpha-keto acid (BCAA) dehydrogenase complex, in a subject. In some aspects the disclosure provides compositions and methods for promoting expression of functional BCKDHB protein, which is the E1-beta subunit of the branched-chain alpha-keto acid (BCAA) dehydrogenase complex, in a subject. In some aspects the disclosure provides compositions and methods for promoting expression of functional BCKDHA and BCKDHB proteins, in a subject. In some embodiments, the disclosure provides methods of treating a subject having Maple Syrup Urine Disease (MSUD).

    COMPOSITIONS AND METHODS FOR TRANSIENT DELIVERY OF NUCLEASES
    119.
    发明申请
    COMPOSITIONS AND METHODS FOR TRANSIENT DELIVERY OF NUCLEASES 审中-公开

    公开(公告)号：US20200231953A1

    公开(公告)日：2020-07-23

    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 ,  A61K48/00 ,  C12N15/90 ,  C07K14/005 ,  C12N7/00 ,  C12N15/11

    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.

Patent Agency Ranking