Methods of reducing expression of X-inactivation escapee genes and autosomal genes
AssigneeThe General Hospital Corporation
InventorsJeannie T. Lee, Hsueh-Ping Chu
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Abstract
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.
Claims10
A method of decreasing expression of an Xi escapee gene, or decreasing expression of Xa genes, in a cell, the method comprising administering to the cell an inhibitory nucleic acid targeting PAR-TERRA.
A method of decreasing expression of autosomal genes in a cell, the method comprising administering to the cell an inhibitory nucleic acid targeting PAR-TERRA or an autosome-specific TERRA, optionally TERRA species originating within the subtelomeric region of an autosome and comprising autosome-specific 5′ sequences, optionally wherein the inhibitory nucleic acid is modified.
A method of decreasing expression of X-linked or autosomal growth control or apoptosis genes in a cell, the method comprising administering to the cell an inhibitory nucleic acid targeting PAR-TERRA, PAR, or TERRA, optionally wherein the inhibitory nucleic acid is modified.
Dependencies are inferred from the claim text.