Enhanced brain distribution of modified aspartoacylase.
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ABSTRACT:
SUBMITTER: Poddar NK
PROVIDER: S-EPMC4252805 | biostudies-literature | 2014 Nov
REPOSITORIES: biostudies-literature
Publications
Enhanced brain distribution of modified aspartoacylase.
Poddar Nitesh K NK Zano Stephen S Natarajan Reka R Yamamoto Bryan B Viola Ronald E RE
Molecular genetics and metabolism 20140712 3
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 ...[more]
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