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.
Study highlights unexpected interactions between innate immune system and neurons, raises questions about additional roles for immune system in the brain with aging.
Whitehead and Broad scientists have developed a gene-silencing tool that could pave the way for a new class of genetic approaches to treat certain diseases.
A study of brain tissue from living adults provides a rare look into the earliest stages of the neurodegenerative disease and highlights cell types involved in plaque production.