A paper recently published in EMBO Molecular Medicine by Sorrentino et al. showed efficacy of a chimeric sulphamidase that was engineered by adding the signal peptide from the highly secreted iduronate-2-sulphatase and the blood-brain barrier (BBB)-binding domain from the Apolipoprotein B. This bioengineered sulphamidase was highly secreted, was efficient in BBB transcytosis, restored enzyme activity in the brain, improved brain pathology, and behavioural phenotype of MPSIIIA mice. These results have potential for therapy of brain disease of MPS-IIIA by enzyme replacement therapy or gene therapy.
Part 16: LYSOSOMAL DISORDERS, chapter 136: The Mucopolysaccharidoses
Posted by Nicola Brunetti-Pierri, MD