The blood-brain barrier (BBB) is the brain’s self-defence mechanism, keeping out bacteria and viruses but allowing in vital nutrients; unfortunately, it also blocks a very high per cent of drugs designed to treat neurological conditions.
Neurological diseases affect the central and peripheral nervous system, which includes the brain, spinal cord and nerves.
Here, we discuss current approaches to delivering drugs into the central nervous system (CNS), focusing on the specific challenges for key therapies like gene-silencing drugs that aim to treat rare neurological diseases. Almost half of the 8,000-10,000 known rare conditions are neurological.
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