Tolerating Enzyme Delivery

Researchers from the University of California, Los Angeles and BioMarin Pharmaceutical Inc. have published in the Journal of Clinical Investigation a strategy for boosting the efficacy of enzyme replacement therapies in lysosomal storage disorders by inducing immune tolerance. Timing and dosing of the tolerance regime will be the major challenges to such an approach.

Lysosomal storage disorders (LSDs) are genetic diseases characterized by absent or malfunctioning lysosomal enzymes that lead to aberrant metabolism of proteins, carbohydrates or lipids in the lysosome. These diseases have varying degrees of severity, but all involve the buildup of enzyme substrate in the lysosome that affects tissue and organ function...