Inside Precision Medicine February 13, 2025
A groundbreaking study has used cutting-edge epigenome editing technologies to advance understanding and potential treatments for Prader-Willi syndrome (PWS), a complex neuroendocrine and neurobehavioral disorder caused by DNA imprinting. The study, published in Cell Genomics, highlights the transformative potential of precision epigenome editing in therapeutic development.
Unlocking epigenetic mysteries
Although extensive research has been conducted on the identity and dynamics of epigenetic marks, the mechanisms driving heritable and allele-specific gene expression changes remain unclear. Scientists have turned to advanced tools, such as CRISPR-Cas9, to manipulate gene expression and epigenetic states in living cells with unprecedented precision.
Using nuclease-deactivated Cas9 (dCas9) fused with transcriptional regulators or epigenome modifiers, researchers can target specific DNA regions to alter chromatin marks, modify DNA methylation,...
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