Trying to outrun her disease, researcher creates powerful epigenetic editor
A powerful epigenetic editor developed by researchers at the Broad Institute aims to combat prion disease, a rare neurodegenerative condition. The tool utilizes CRISPR gene editing to potentially treat this fatal disease.
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A researcher at the Broad Institute, Sonia Vallabh, along with her husband Eric Minikel, has developed a powerful epigenetic editor in mice to combat prion disease. Prion disease is a rare form of neurodegeneration that Vallabh's mother succumbed to at age 51, and Vallabh herself is at risk. The disease is caused by a genetic mutation leading to the production of misfolded prions that are toxic to neurons. Vallabh and Minikel have been exploring using CRISPR gene editing to repair or remove the mutant gene responsible for the disease. Their work aims to find a treatment for this insidious disease that can be fatal if left untreated. By creating this gene editing tool, they hope to pave the way for potential treatments for prion disease.
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