Abstract
The development of epigenetic editing technology enables dissection of functional significance of epigenetic events associated with disease. Fused with DNA-targeting proteins, such as the catalytically inactivated Cas9 protein, enzymes catalyzing DNA methylation and histone modification can be recruited to specific regulatory genomic regions to mediate epigenetic editing. In addition, the specific degradation of noncoding RNA by antisense oligonucleotide is another type of epigenetic mechanism. Application of epigenetic editing technology is particularly promising for the study of imprinting disorders and X-linked disease, because while one allele of the disease-related gene loses function, either by mutation or epimutation, a wild-type allele on the other chromosome is silenced by epigenetic mechanisms. Thus, reactivating the wild-type copy through epigenetic editing has therapeutic potential for such developmental disorders. Finally, challenges of epigenetic editing, including off-target effects, epigenetic efficacy, phenotypical potency, and persistence, will be discussed.
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