A single-letter edit in DNA reduces levels of the disease-causing prion protein in the brain and could lead to a preventative, one-time treatment for the deadly neurodegenerative disorder.
Whitehead and Ó³»´«Ã½ scientists have developed a gene-silencing tool that could pave the way for a new class of genetic approaches to treat certain diseases.
The gene-editing approach uses prime editors and evolved enzymes called recombinases, and could pave the way to effective one-size-fits-all gene therapies for diseases such as cystic fibrosis.