Enhanced brain distribution of modified aspartoacylase.

[canavan disease]

Canavan disease is a fatal neurological disorder caused by defects in the gene that produces the enzyme aspartoacylase . Enzyme replacement therapy can potentially be used to overcome these defects if a stable enzyme form that can gain access to the appropriate neural cells can be produced . Achieving the proper cellular targeting requires a modified form of aspartoacylase that can traverse the blood -brain barrier . A PEGylated form of aspartoacylase that shows dramatic enhancement in brain tissue access and distribution has been produced . While the mechanism of transport has not yet been established , this modified enzyme is significantly less immunogenic than unmodified aspartoacylase . These improved properties set the stage for more extensive enzyme replacement trials as a possible treatment strategy .