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METHODS OF REDUCING EXPRESSION OF X-INACTIVATION ESCAPEE GENES AND AUTOSOMAL GENES
METHODS OF REDUCING EXPRESSION OF X-INACTIVATION ESCAPEE GENES AND AUTOSOMAL GENES
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机译:减少X-失活超视基因和常染色体基因表达的方法
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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.
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