Abstract
Neurovascular disorders are difficult to treat due to the blood–brain barrier (BBB), which prevents delivery of most drugs from the blood into the brain. Gene therapy can potentially overcome this barrier, but classical vectors are neither efficient nor specific enough to provide long‐lasting treatment or avoid off‐target effects. In this issue of EMBO Molecular Medicine , Korbelin et al (2016) describe an engineered adeno‐associated virus (AAV) with exceptional tropism for the brain that restores a normal phenotype in a mouse model of incontinentia pigmenti (IP), a severe human genetic disorder of brain vasculature.
Highlights
Neurovascular disorders are difficult to treat due to the blood–brain barrier (BBB), which prevents delivery of most drugs from the blood into the brain
Neurovascular disorders are a heterogeneous group of pathologies that display a common characteristic: they are difficult to treat with traditional pharmacological approaches. This is largely due to restraints imposed by the BBB that limit drug distribution to neurons and glial cells of the central nervous system (CNS) upon systemic administration
Astrocyte glial cells envelop the outer surface of small blood vessels in the CNS and impose further restrictions
Summary
Neurovascular disorders are difficult to treat due to the blood–brain barrier (BBB), which prevents delivery of most drugs from the blood into the brain. In the brain and spinal cord, endothelial cells lining the entire inner surface of blood vessels are linked to one another by tight junctions, which inhibit transport of most compounds across the BBB. Astrocyte glial cells envelop the outer surface of small blood vessels in the CNS and impose further restrictions.
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