## 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
✦ LIBER ✦
Pharmacokinetics and Safety in Rhesus Monkeys of a Monoclonal Antibody-GDNF Fusion Protein for Targeted Blood-Brain Barrier Delivery
✍ Scribed by William M. Pardridge; Ruben J. Boado
- Publisher
- Springer US
- Year
- 2009
- Tongue
- English
- Weight
- 210 KB
- Volume
- 26
- Category
- Article
- ISSN
- 0724-8741
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## Abstract Protein therapeutics may be delivered across the blood–brain barrier (BBB) by genetic fusion to a BBB molecular Trojan horse. The latter is an endogenous peptide or a peptidomimetic monoclonal antibody (MAb) against a BBB receptor, such as the insulin receptor or the transferrin recepto