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    Methods of reducing expression of X-inactivation escapee genes and autosomal genes
    2.
    发明授权
    Methods of reducing expression of X-inactivation escapee genes and autosomal genes 有权

    公开(公告)号：US10519444B2

    公开(公告)日：2019-12-31

    申请号：US15780509

    申请日：2016-12-01

    申请人： The General Hospital Corporation

    发明人： Jeannie T. Lee ,  Hsueh-Ping Chu

    IPC分类号： C12N15/113 ,  A61K31/713 ,  A61K45/06

    摘要： Inhibitory nucleic acids, e.g., antisense oligonucleotides (ASO) against PAR-TERRA RNA or other chromosome-specific TERRA transcripts (i.e., inclusive of chromosome-specific subtelomeric sequences), and methods of use thereof to downregulate expression of escapee genes on the inactive X chromosome, expression from the active X chromosome, subtelomeric autosomal loci (e.g., FSHD locus), or expression of autosomal genes involved in growth control and apoptosis, e.g., in cells and subjects with supernumerary X chromosomes and/or cancer and other human diseases.

    CCCTC-Binding Factor (CTCF) RNA Interactome
    3.
    发明申请
    CCCTC-Binding Factor (CTCF) RNA Interactome 审中-公开

    公开(公告)号：US20190309291A1

    公开(公告)日：2019-10-10

    申请号：US15952679

    申请日：2018-04-13

    申请人： The General Hospital Corporation

    发明人： Jeannie T. Lee ,  Johnny Kung ,  Barry Kesner

    IPC分类号： C12N15/113

    摘要： This invention relates to methods and compositions for selectively reactivating or downregulating certain genes, e.g., genes regulated by zinc-finger protein CCCTC-binding factor (CTCF) on autosomes (e.g., imprinted genes, tumor suppressors, cancer) and the inactive X chromosome (Xi), e.g., genes associated with X-linked diseases, e.g., RettSyndrome, Factor VIII or IX deficiency, Fragile X Syndrome, Duchenne muscular dystrophy, and PNH, in heterozygous females carrying a mutated allele, in addition to a functional wildtype or hypomorphic allele.

    Methods for reactivating genes on the inactive X chromosome
    10.
    发明授权
    Methods for reactivating genes on the inactive X chromosome 有权

    公开(公告)号：US10961532B2

    公开(公告)日：2021-03-30

    申请号：US15565060

    申请日：2016-04-06

    申请人： The General Hospital Corporation

    发明人： Jeannie T. Lee ,  Anand Minajigi ,  Lieselot Carrette

    IPC分类号： C12N15/113 ,  C12Q1/6883 ,  C12N9/10 ,  C12N9/90

    摘要： Methods for reactivating genes on the inactive X chromosome that include administering one or both of a DNA methyltransferase (DNMT) Inhibitor and/or a topoisomerase inhibitor, e.g., etoposide and/or 5-azacytidine (aza), optionally in combination with an inhibitor of XIST RNA and/or an Xist-interacting protein, e.g., a chromatin-modifying protein, e.g., a small molecule or an inhibitory nucleic acid (such as a small inhibitory RNA (siRNAs) or antisense oligonucleotide (ASO)) that targets XIST RNA and/or a gene encoding an Xist-interacting protein, e.g., a chromatin-modifying protein.