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    COMPOSITIONS USEFUL IN TREATMENT OF RETT SYNDROME
    3.
    发明申请
    COMPOSITIONS USEFUL IN TREATMENT OF RETT SYNDROME 审中-公开

    公开(公告)号：WO2022094078A1

    公开(公告)日：2022-05-05

    申请号：PCT/US2021/057047

    申请日：2021-10-28

    申请人： THE TRUSTEES OF THE UNIVERSITY OF PENNSYLVANIA

    发明人： WILSON, James, M. ,  SCHMID, Ralf

    IPC分类号： C12N15/86 ,  A61P25/00 ,  A61K48/00

    摘要： Provided is a recombinant adeno-associated virus (rAAV) having an AAV capsid and a vector genome which comprises a nucleic acid sequence encoding a functional human methyl-CpG-binding protein 2 (hMECP2). Also provided is a production system useful for producing the rAAV, a pharmaceutical composition comprising the rAAV, and a method of treating a subject having Rett Syndrome, or ameliorating symptoms of Rett Syndrome, or delaying progression of Rett Syndrome via administrating an effective amount of the rAAV to a subject in need thereof.

    COMPOSITIONS AND METHODS FOR REDUCING CHOLESTEROL LEVELS
    6.
    发明申请
    COMPOSITIONS AND METHODS FOR REDUCING CHOLESTEROL LEVELS 审中-公开

    公开(公告)号：WO2021080975A1

    公开(公告)日：2021-04-29

    申请号：PCT/US2020/056470

    申请日：2020-10-20

    申请人： THE TRUSTEES OF THE UNIVERSITY OF PENNSYLVANIA

    发明人： WANG, Lili ,  WILSON, James, M. ,  WANG, Qiang

    IPC分类号： A61P3/06 ,  C07K14/705 ,  C12N15/86

    摘要： A recombinant vector having an expression cassette comprising an engineered nucleic acid sequence encoding a functional human low-density lipoprotein receptor (hLDLR) is provided. The functional hLDLR protein is a wild type or a modified hLDLR comprising three amino acid substitutions (i.e., L318D, K809R and C818A). A recombinant adeno-associated viral particle (rAAV) is also provided comprising a capsid and a vector genome packaged therein, wherein the vector genome comprises an engineered nucleic acid sequence encoding a functional hLDLR. The capsid may be an AAV8 capsid, an AAV3B capsid, or an AAV38 variant capsid. Further provided are pharmaceutical compositions comprising a recombinant vector or an rAAV and a pharmaceutical acceptable carrier, as well as uses and methods using the recombinant vector, rAAV or composition in lowering cholesterol and/or treating familial hypercholesterolemia.

    COMPOSITIONS USEFUL IN TREATMENT OF RETT SYNDROME
    7.
    发明申请
    COMPOSITIONS USEFUL IN TREATMENT OF RETT SYNDROME 审中-公开

    公开(公告)号：WO2020219766A1

    公开(公告)日：2020-10-29

    申请号：PCT/US2020/029642

    申请日：2020-04-23

    申请人： THE TRUSTEES OF THE UNIVERSITY OF PENNSYLVANIA

    发明人： WILSON, James, M. ,  SCHMID, Ralf

    IPC分类号： C12N15/117 ,  C12N15/861 ,  A61P25/00

    摘要： Provided is a recombinant adeno-associated virus (rAAV) having an AAV capsid and a vector genome which comprises a nucleic acid sequence encoding a functional human methyl-CpG-binding protein 2 (hMECP2). Also provided is a production system useful for producing the rAAV, a pharmaceutical composition comprising the rAAV, and a method of treating a subject having Rett Syndrome, or ameliorating symptoms of Rett Syndrome, or delaying progression of Rett Syndrome via administrating an effective amount of the rAAV to a subject in need thereof.

    RECOMBINANT ADENO-ASSOCIATED VIRUS FOR TREATMENT OF GRN-ASSOCIATED ADULT-ONSET NEURODEGENERATION
    9.
    发明申请
    RECOMBINANT ADENO-ASSOCIATED VIRUS FOR TREATMENT OF GRN-ASSOCIATED ADULT-ONSET NEURODEGENERATION 审中-公开

    公开(公告)号：WO2020172490A1

    公开(公告)日：2020-08-27

    申请号：PCT/US2020/019149

    申请日：2020-02-21

    申请人： THE TRUSTEES OF THE UNIVERSITY OF PENNSYLVANIA

    发明人： HINDERER, Christian ,  MILLER, Nimrod ,  WILSON, James, M.

    IPC分类号： A61K48/00 ,  C12N15/861

    摘要： A recombinant AAV (rAAV) suitable for use in treating adult onset neurodegeneration caused by granulin (GRN) haploinsufficiency, such as progranulin (PGRN) - related frontotemporal dementia (FTD), is provided. The rAAV comprises (a) an adeno-associated virus 1 capsid, and (b) a vector genome packaged in the AAV capsid, said vector genome comprising AAV inverted terminal repeats, a coding sequence for human progranulin, and regulatory sequences which direct expression of the progranulin. Also provided are a method for treating a human patient with PGRN -FTD and other adult onset neurodegeneration caused by granulin (GRN) haploinsufficiencies, comprising delivering to the central nervous system (CNS) a recombinant adeno-associated virus (rAAV) having an adeno-associated virus 1 (AAV1) capsid, said rAAV further comprising a vector genome packaged in the AAV capsid, said vector genome comprising AAV inverted terminal repeats, a coding sequence for human progranulin, and regulatory sequences which direct expression of the progranulin.