HealthBiotechDispatchAustralian scientists switch on silenced genes in Prader-Willi syndrome
Melbourne researchers at WEHI have found a way to switch back on silenced genes in Prader-Willi syndrome, potentially leading to treatments that target the cause, not just symptoms.
"Most people can't conceive what daily life is like for a person living with Prader-Willi syndrome," said Kathlene Jones, CEO of the Prader-Willi Research Foundation Australia.
Researchers at Melbourne's Walter and Eliza Hall Institute have found a way to reactivate genes that are silenced in Prader-Willi syndrome, in what WEHI calls a world first.
The team used CRISPR gene editing and stem-cell technologies to remove a protein called SMCHD1, which acts as a genetic brake keeping key brain-function genes switched off.
Lead researcher Professor Marnie Blewitt says they now have the strongest evidence the approach can restore gene activity without disrupting the rest of the body, paving the way for future treatments.
The WEHI team used patient-derived cells and animal models and found removing the SMCHD1 protein restored gene activity safely, which researchers say is the strongest evidence yet for a future…
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