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    COMPOSITIONS AND METHODS FOR TARGETED INACTIVATION OF HIV CELL SURFACE RECEPTORS
    73.
    发明公开
    COMPOSITIONS AND METHODS FOR TARGETED INACTIVATION OF HIV CELL SURFACE RECEPTORS 审中-公开
    组合物和有针对性的禁止艾滋病病毒的细胞表面受体方法

    公开(公告)号：EP2099911A2

    公开(公告)日：2009-09-16

    申请号：EP08727618.4

    申请日：2008-01-11

    申请人： Yale University

    发明人： GLAZER, Peter, M. ,  BINDRA, Ranjit ,  SCHLEIFMAN, Erica, B.

    IPC分类号： C12N15/11 ,  C12N15/10 ,  A61P31/18

    CPC分类号： C12N15/1138 ,  C12N15/111 ,  C12N2310/15 ,  C12N2310/3181 ,  C12N2310/3519 ,  C12N2320/30

    摘要： Compositions for targeted mutagenesis of cell surface receptors for HIV and methods of their use are provided herein. The compositions include triplex-forming molecules that bind to duplex DNA in a sequence specific manner at target sites to form triple-stranded structures. The triplex-forming molecules can be triplex-forming oligonucleotides (TFOs) or peptide nucleic acids (PNAs). The triplex-forming molecules are useful to induce site-specific homologous recombination in mammalian cells when used in combination with donor oligonucleotides. The triplex-forming molecules target sites within or adjacent to genes that encodes cell surface receptors for human immunodeficiency virus (HIV). This binding stimulates homologous recombination of a donor oligonucleotide to cause mutations in HIV cell surface receptor genes that result in one or more deficiencies in the ability of the encoded receptor to bind to HIV and allow its transport into the cell. Methods for ex vivo and in vivo prophylaxis and therapy of HIV infection using the disclosed compositions are also provided.