A Potential Gene Therapy For Mucopolysaccharidosis Type IIIA

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 glycosaminoglycans. Over time, the glycosaminoglycans collect in the body and cause damage, particularly in the brain. In this issue of the Journal of Clinical Investigation, Fatima Bosch and colleagues at Universitat Autonoma de Barcelona in Spain developed a form of gene therapy to replace the enzyme that is missing in MPSIIIA... via Health News from Medical News Today Read More Here..

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