A gene therapy delivery platform reported a strategy to reach brain targets in mice by engineering AAV capsids and using a glymphatic delivery approach. Researchers at University of Rochester Medicine and the University of Copenhagen reported in Nature Biotechnology that the engineered AAV vectors preferentially infected human glial progenitor cells in vivo, aiming to improve on the twin hurdles of blood-brain barrier crossing and off-target delivery. If the targeting holds in later models, the platform could support expanded development of cell and gene therapies for diseases where glial pathology is central.
Get the Daily Brief