Most pharmaceuticals don't reach the brain, leaving cognitive impairments due to diseases like mucopolysaccharidoses (MPS) hard to treat. Now, a...

A recent proof-of-concept study from the Sontheimer lab at the University of Massachusetts Chan Medical School uses all-in-one, self-inactivating AAV...

CRISPR-based blood cell therapies are advancing through clinical trials, with promising results stacking up for certain rare genetic diseases and some...