Intrathecal enzyme replacement therapy: successful treatment of brain disease via the cerebrospinal fluid
P Dickson, M McEntee, C Vogler, S Le, B Levy… - Molecular genetics and …, 2007 - Elsevier
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 iduronidase (rhIDU) in canine
mucopolysaccharidosis I (MPS I, Hurler syndrome), a lysosomal storage disorder with 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 iduronidase (rhIDU) in canine
mucopolysaccharidosis I (MPS I, Hurler syndrome), a lysosomal storage disorder with brain …
