New platform combines precision gene targeting with brain-wide delivery

Article excerpt

A new study describes a gene therapy strategy that uses the brain's own glymphatic transport system to distribute engineered viral vectors throughout the brain. The approach addresses two major challenges in neurological medicine, reaching therapeutic targets behind the blood-brain barrier and limiting unwanted effects elsewhere in the body, and could pave the way for new treatments for diseases including multiple sclerosis, Huntington's disease and rare childhood white matter disorders.