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New health and medicine research from University of Bonn, Department of Physiology discussed

Published in Gene Therapy Weekly, July 15th, 2010

Researchers detail in 'Metachromatic leukodystrophy--an update,' new data in health and medicine. According to a study from Germany, "Metachromatic leukodystrophy (MLD) is a rare lysosomal sphingolipid storage disorder, caused by a deficiency of arylsulfatase A (ASA). It is inherited in an autosomal recessive way, among Caucasians three causing alleles are frequent."

"Demyelination is the hallmark of MLD. Interest in the disease has increased as therapeutic options such as stem cell transplantation, enzyme replacement and gene therapy are topics of current research. A late-infantile (onset before 3 years of age), a juvenile form (onset before 16 years) and an adult form...

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