Posted on Jul 2, 2024
CHARMed collaboration creates a potent therapy candidate for fatal prion diseases
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A new gene-silencing tool shows promise as a future therapy against prion diseases and paves the way for new approaches to treating disease.
Vallabh and her husband, Eric Minikel, switched careers and became researchers after they learned that Vallabh carries a disease-causing version of the prion protein gene and that there is no effective therapy for fatal prion diseases. The two now run a lab at the Broad Institute, where they are working to develop drugs that can prevent and treat these diseases, and their deadline for success is not based on grant cycles or academic expectations but on the ticking time bomb in Vallabh’s genetic code.
Vallabh and her husband, Eric Minikel, switched careers and became researchers after they learned that Vallabh carries a disease-causing version of the prion protein gene and that there is no effective therapy for fatal prion diseases. The two now run a lab at the Broad Institute, where they are working to develop drugs that can prevent and treat these diseases, and their deadline for success is not based on grant cycles or academic expectations but on the ticking time bomb in Vallabh’s genetic code.
CHARMed collaboration creates a potent therapy candidate for fatal prion diseases
Posted from news.mit.edu
Medical
Research
Health
Science
Edited 5 mo ago
Posted 5 mo ago
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I am not very familiar with Gene Therapy, but I take it when cells are edited they only reproduce the edited Gene.
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Lt Col Charlie Brown
Yes, that's true. It works particularly well when there's an enzyme that needs to be regulated to cure a condition. The gene can be edited to produced more or less of it as the condition warrants.
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