Normalization and Improvement of CNS Deficits in Mice With Hurler Syndrome After Long-term Peripheral Delivery of BBB-targeted Iduronidase

Mucopolysaccharidosis type I (MPS I) is a progressive lysosomal storage disorder with systemic and central nervous system (CNS) involvement due to deficiency of α-l-iduronidase (IDUA). We previously identified a receptor-binding peptide from apolipoprotein E (e) that facilitated a widespread deliver...

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Detalhes bibliográficos
Publicado no:Mol Ther
Main Authors: El-Amouri, Salim S, Dai, Mei, Han, Jing-Fen, Brady, Roscoe O, Pan, Dao
Formato: Artigo
Idioma:Inglês
Publicado em: Nature Publishing Group 2014
Assuntos:
Original Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC4429694/
https://ncbi.nlm.nih.gov/pubmed/25088464
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mt.2014.152
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