This is a news story, published by MIT News, that relates primarily to CRISPRoff news.
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Biomedical Research member Jonathan WeissmanMIT News
•82% Informative
A set of molecular tools called CHARMs can turn off disease-causing genes such as the prion protein gene.
The tools still have many hurdles to pass before the researchers will know if they work as therapeutics.
CHARMs are an elegant solution to the problem of silencing disease genes.
CRISPRoff is a tool that silences the targeted gene by adding chemical tags that prevent the gene from being transcribed, or read into RNA , and so from being expressed as protein.
The tool was designed to be small enough to be packaged and delivered to specific cells in the body.
It was also designed to minimize the risk of silencing the wrong genes or causing side effects.
A new project from the Broad Institute has brought the CHARM technology to clinical trials for brain-wide gene therapy.
CHARMs are currently being tested as therapeutics in mice.
The team is now fine-tuning their tool to make it more effective, safer, and easier to produce at scale.
CHARMs still have a way to go before they might become a viable medical option for people with prion diseases.
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