METHODS FOR REACTIVATING GENES ON THE INACTIVE X CHROMOSOME

摘要

Methods for reactivating genes on the inactive X chromosome that include administering an inhibitor of XIST RNA and an inhibitor of 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.