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Design of ICAM-1-Targeting Strategies for Brain Delivery of Lysosomal Therapies

✍ Scribed by Silvia Muro; Janet Hsu; Tridib Bhowmick; Carmen Garnacho; Silvia Muro


Book ID
116989202
Publisher
Elsevier Science
Year
2011
Tongue
English
Weight
56 KB
Volume
102
Category
Article
ISSN
1096-7192

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## Abstract Mucopolysaccharidosis Type I, Hurler's Syndrome, is a lysosomal storage disorder that affects the brain. The missing enzyme, α‐L‐iduronidase (IDUA), does not cross the blood‐brain barrier (BBB). To enable BBB transport of the enzyme, human IDUA was fused to the carboxyl terminus of the