Alpha-L-iduronidase

Hartung, S. D., Frandsen, J. L., Pan, D., Koniar, B. L., Graupman, P., Gunther, R., Low, W. C., Whitley, C. B. and Mclvor, R. S. (2004). Correction of metabolic, craniofacial, and neurologic abnormalities in MPS I mice treated at birth with adeno-associated virus vector transducing the human alpha-L-iduronidase gene. Mol. Ther. 9, 866-875. 

Genetically corrected autologous stem cells engraft, but host immune responses limit their utility in canine alpha-L-iduronidase deficiency. Blood 93, 1895-1905. 

Meertens, L., Zhao, Y., Rosic-Kablar, S., Li, L., Chan, K., Dobson, H., Gartley, C., Lutzko, C., Hopwood, J., Kohn, D., Kruth, S., Hough, M. R. and Dube, I. D. (2002). In utero injection of alpha-L-iduronidase-carrying retrovirus in canine mucopolysaccharidosis type I Infection of multiple tissues and neonatal gene expression. Hum. Gene Ther. 13, 1809-1820. 

Wraith, J. E., Clarke, L. A., Beck, M., Kolodny, E. H., Pastores, G. M., Muenzer, J., Rapoport, D. M., Berger, K. I., Swiedler, S. J., Kakkis, E. D., Braakman, T., Chadbourne, E., Walton-Bowen, K. and Cox, G. F. (2004). Enzyme replacement therapy for mucopolysaccharidosis I A randomized, double-blinded, placebo-controlled, multinational study of recombinant human alpha-L-iduronidase (laronidase). J. Pediatr. 144, 581-588. 

Shull RM, Munger RJ, Spellacy E, Hall CW, Constantopoulos G, Neufeld EF. Canine alpha-L-iduronidase deficiency. A model of mucopolysaccharidosis I. Am J Pathol 1982 109(2) 244-8. 

Spellacy E, Shull RM, Constantopoulos G, Neufeld EF. A canine model of human alpha-l-iduronidase deficiency. Proc Natl Acad Sci USA 1983 80(19) 6091-5. 

Alpha -L- iduronidase MPSI BioMarin, Genzyme Phase 111... 

USA laronidase enzyme Aldurazyme Recombinant human 8-L-histidine alpha-L-iduronidase expressed in CHO cells Polysaccharide- sis... 

Laronidase rh-alpha-L-iduronidase precursor rh-IDU (Aldurazyme) 69.9 0.24-0.60 1.5-3.6 IV infusion (1.0) Mucopolysaccaridosis 1 (Hurler and Scheie syndromes)... 

---