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Chen A, Vogler C, McEntee M, Hanson S, Ellinwood MN, Jens J, Snella E, Passage M, Le S, Guerra C, Dickson P. Glycosaminoglycan storage in neuroanatomical regions of mucopolysaccharidosis I dogs following intrathecal recombinant human iduronidase. APMIS 2011; 119: 513–21.
Intrathecal (IT) recombinant human α‐l‐iduronidase (rhIDU) has been shown to reduce mean brain glycosaminoglycans (GAGs) to normal...
Treatment of brain disease with recombinant proteins is difficult due to the blood–brain barrier. As an alternative to direct injections into the brain, we studied whether application of high concentrations of therapeutic enzymes via intrathecal (IT) injections could successfully drive uptake across the ependyma to treat brain disease. We studied IT enzyme replacement therapy with recombinant human...
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