Switching on healthy silent genes reveals new way to treat Prader-Willi syndrome
WEHI researchers have discovered a way to release a genetic 'brake' that silences healthy genes in Prader-Willi syndrome, opening a potential new path to treating the condition.
A team of researchers from WEHI has developed a method to reactivate dormant genes in individuals with Prader-Willi syndrome, a complex genetic disorder affecting millions worldwide. Prader-Willi syndrome disrupts brain function, leading to developmental delays, intellectual disability, and an uncontrollable urge to eat, often resulting in obesity.
The protein SMCHD1, discovered at WEHI in 2008, plays a crucial role in silencing vital genes responsible for normal development and brain function. By removing SMCHD1, researchers were able to lift this genetic brake, allowing these dormant genes to switch back on. This groundbreaking finding, published in Science Advances, offers a potential new treatment approach that targets the root cause of the syndrome, rather than merely managing its symptoms.
The study's success marks a significant milestone in a long-standing research journey at WEHI, with the ultimate goal of developing effective medicines for those affected by Prader-Willi syndrome.
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