Stanford researchers have created a CRISPR-based technology to target RNA in neurons, enabling repair processes for genetic diseases. The method addresses key challenges in Cas9 delivery and DNA repair. It reveals unique genome editing rules in neurons, paving the way for targeted therapies.

Researchers developed the first experimental mRNA-based therapy for ISG15 deficiency, a rare genetic mutation providing near-universal viral immunity, as reported on September 9, 2025.

Illumina introduced an AI tool aiding in the diagnosis of rare diseases. This innovation enhances genetic analysis for complex conditions. It was highlighted in biotech news updates.