Targeting white matter astrocytes with a Gfap-disruptive Cas9 nuclease benefits a murine model of Alexander disease.
Astrocytes are intriguing therapeutic targets due to their crucial role in initiating and promoting demyelination in the central nervous system. By efficient on-target editing, we can permanently disrupt detrimental genes and restore normal physiological processes. Here, we have optimized an AAV platform to deliver the...
Anne E. J. Hillen, I. Capuano, Barbara Gega et al.
· Molecular Therapy · 0 citations