Intrathecal enzyme replacement therapy reduces lysosomal storage in the brain and meninges of the canine model of MPS I
E Kakkis, M McEntee, C Vogler, S Le, B Levy… - Molecular genetics and …, 2004 - Elsevier
Enzyme replacement therapy (ERT) has been developed for several lysosomal storage
disorders, including mucopolysaccharidosis I (MPS I), and is effective at reducing lysosomal
storage in many tissues and in ameliorating clinical disease. However, intravenous ERT
does not adequately treat storage disease in the central nervous system (CNS), presumably
due to effects of the blood–brain barrier on enzyme distribution. To circumvent this barrier,
we studied whether intrathecal (IT) recombinant human α-l-iduronidase (rhIDU) could …
disorders, including mucopolysaccharidosis I (MPS I), and is effective at reducing lysosomal
storage in many tissues and in ameliorating clinical disease. However, intravenous ERT
does not adequately treat storage disease in the central nervous system (CNS), presumably
due to effects of the blood–brain barrier on enzyme distribution. To circumvent this barrier,
we studied whether intrathecal (IT) recombinant human α-l-iduronidase (rhIDU) could …
