New Study Examines How Drugs Can Regulate Gene Expression. Important Steps Toward Reactivating the Maternal Allele

“Isn’t there a way to just ‘turn on’ the silent PWS genes on chromosome 15?” It's a common question amongst the PWS community. The PWS genes on the maternal chromosome 15 are present but silenced in all people with PWS, regardless of genetic subtype. In theory, if you could ‘turn on’ those maternal PWS genes, you might be able to produce a therapeutic effect. FPWR is fortunate to have funded several projects aimed exploring this question. The findings of one of those projects has just been published in the Proceedings of the National Academy of Sciences (Powell, 2013), and it investigates the molecular mechanisms by which drugs might be used to regulate/re-activate genes in the Angelman’s/PWS region. The findings advance the basic understanding of PWS gene regulation and also have potential therapeutic implications for both PWS and Angelman’s syndrome (AS). Learn more about this exciting development on our Research Blog.