Selective reactivation of genes on the inactive X chromosome

摘要

Methods and compositions for selectively reactivating genes on the inactive X chromosome (Xi) in a locus-specific manner, e.g., genes associated with X-linked diseases, e.g., Rett Syndrome, Factor VIII or IX deficiency, Fragile X Syndrome, Duchenne muscular dystrophy, and PNH, in heterozygous females.

主权项

1. A method of activating an inactive X-linked allele in a cell, the method comprising administering to the cell
an inhibitory oligonucleotide targeting a PRC2 binding site in a region of a gene encoding the allele on the X Chromosome that is marked concurrently by H3K4me3 and H3K27me3 and coincides with a CpG island.