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    OPTIMIZED GENE THERAPY FOR TARGETING MUSCLE IN MUSCLE DISEASES
    1.
    发明公开
    OPTIMIZED GENE THERAPY FOR TARGETING MUSCLE IN MUSCLE DISEASES 审中-公开

    公开(公告)号：US20230227515A1

    公开(公告)日：2023-07-20

    申请号：US17786721

    申请日：2020-12-21

    申请人： RESEARCH INSTITUTE AT NATIONWIDE CHILDREN'S HOSPITAL

    发明人： Paul Taylor Martin

    IPC分类号： C07K14/47 ,  A61K48/00 ,  C12N15/86 ,  A61P21/00

    CPC分类号： C07K14/4707 ,  A61K48/005 ,  C12N15/86 ,  A61P21/00

    摘要： The disclosure provides gene therapy vectors, such as adeno-associated virus (AAV), optimized for delivering a transgene to muscles. The optimized vectors contain constitutive or a muscle-specific promoter to deliver whole body or skeletal/heart muscle-specific transgene expression, respectively, in combination with a transgene cDNA to replace the gene mutation found in a muscle disease with a normal copy of the gene, an internal ribosomal entry site (IRES) to allow for production of a second protein from the same transcript, and a muscle growth factor, to build new muscle growth and strength. For example, the invention provides The disclosure provides gene therapy vectors, such as recombinant adeno-associated vims (rAAV), designed for treatment of GNE myopathy in which the rAAV expresses UDP-GlcNAc-epimerase/ManNAc-6 alone or in combination with a muscle growth factor or muscle transdifferentation factor. The provided AAV replace the mutated GNE gene expression while expressing proteins that stimulate muscle growth.