Bypassing the blood–brain barrier: Delivering genetic medicines directly to the brain parenchyma or cerebrospinal fluid
10 Sept 2026
Blood Brain Barrier
- The relative advantages and limitations of CSF and intraparenchymal administration compared with intravenous delivery
- How therapeutic molecules distribute through the brain’s dense extracellular matrix and interstitial space
- How molecules cross the CSF-brain interface, including the ependymal layer, and the depth to which they penetrate brain tissue
- Recent clinical progress in direct-to-brain delivery, including AMT-130 for Huntington’s disease, and advances in administration procedures
- Patient experience, safety considerations, and potential side effects associated with CSF and intraparenchymal administration
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