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A Potential Gene Therapy For Mucopolysaccharidosis Type IIIA

(MPSIIIA) is a metabolic disorder in which the body is missing an enzyme that is required to break down long chains of sugars known as . Over time, the collect in the body and cause damage, particularly in the brain.

In this issue of the , and colleagues at Universitat Autonoma de Barcelona in Spain developed a form of to replace the enzyme that is missing in MPSIIIA. By injecting the replacement gene into the the that surrounds the brain and spinal cord, Bosch and colleagues found that they could successfully deliver a replacement gene to the brain in mice and dogs.

This study demonstrates that gene therapy can be delivered to the brain through the cerebrospinal fluid and suggests that this approach could potentially be used as a therapy for MPSIIIA.

TITLE: Whole body correction of Mucopolysaccharidosis IIIA by intra-cerebrospinal fluid gene therapy



Journal of Clinical Investigation