The system - known as coupled histone tail for autoinhibition release of methyltransferase (CHARM) - changes the 'epigenome', a collection of chemical tags that are attached to DNA and which affect gene activity. In mice, CHARM silenced the gene that produces the disease-causing proteins in most neurons across the brain without altering the gene sequence.
Prion diseases are caused by misfolded prion proteins (PrPs), which clump together and destroy neurons. This can lead to conditions such as fatal familial insomnia - a rare genetic disease that prevents people from sleeping and results in death. Although prion diseases are incurable, drugs known as antisense oligonucleotides (ASOs) have shown some promise.
This system is the first step towards developing a safe and effective 'one and done' treatment for reducing the levels of harmful proteins that cause prion disease, says Madelynn Whittaker, a bioengineer at the University of Pennsylvania in Philadelphia.
#prion-diseases #molecular-editing-tool #epigenome-modification #neurodegenerative-disorders #gene-silencing
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