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Correction of murine mucopolysaccharidosis type IIIA central nervous system pathology by intracerebroventricular lentiviral‐mediated gene delivery

Mucopolysaccharidoses (MPS) are inborn metabolic disorders caused by a deficiency of glycosaminoglycan degrading enzymes. Although intravenous enzyme replacement therapy is a viable approach for the treatment of non‐neuronopathic forms of MPS, its effectiveness in the central nervous system (CNS) is limited by the blood–brain barrier. Alternatively, enzyme replacement therapies and other therapies that directly target the brain represent approaches that circumvent the blood–brain barrier and, in the case of gene therapies, are intended to negate the need for repetitive dosing.