Charm creates a potent therapy candidate for fatal prion diseases
A collaboration between Broad Institute and Whitehead Institute developed CHARM, a gene-silencing tool for prion diseases. CHARMs target disease-causing genes through epigenetic editing, showing promise in mice with low toxicity. Researchers aim to optimize CHARMs for clinical trials.
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A collaboration between the Broad Institute of MIT and Harvard and the Whitehead Institute has led to the development of a promising gene-silencing tool named CHARM, which shows potential as a therapy for fatal prion diseases. Led by researchers Sonia Vallabh and Jonathan Weissman, the team has created CHARMs that can turn off disease-causing genes like the prion protein gene, offering a new approach to treating neurodegenerative diseases. Unlike conventional drugs that target proteins, CHARMs work by epigenetic editing to silence the gene responsible for producing the faulty protein. By utilizing a smaller zinc finger protein and recruiting the cell's own machinery, CHARMs have shown effectiveness in silencing the prion protein gene in mice with low toxicity and limited off-target effects. The researchers are now focusing on optimizing CHARMs for clinical trials, aiming to develop a safe and scalable therapy for genetic diseases like prion diseases. The innovative approach and collaborative effort have accelerated the translation of basic research into potential therapeutic applications in a relatively short timeframe.
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15 commentsPrions are both 100% fatal and virtually indestructible. It's like some sort of glitch universal kill-switch biology happened upon encoded by hell itself.
Also they say that the gene is turned off permanently, but is that really so? I thought epigenetic changes gradually revert themselves - though I suppose that just means you have to reapply the treatment every n years.
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