Antibodies to recombinant human alpha-L-iduronidase prevent disease correction in cortical bone in MPS I mice

Mucopolysaccharidosis I (MPS I) is a lysosomal storage disorder caused by deficiency of the enzyme α-l-iduronidase (IDUA). Failure of enzyme replacement therapy (ERT) to treat skeletal disease may be due to development of anti-IDUA antibodies, found previously to alter tissue distribution of ERT in...

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Hlavní autoři:	Sarah C. Hurt, Steven Q. Le, Shih-hsin Kan, Quang D. Bui, Michael D. Brodt, Patricia I. Dickson
Médium:	Artigo
Jazyk:	Inglês
Vydáno:	Elsevier 2025-03-01
Edice:	Molecular Therapy: Methods & Clinical Development
Témata:	Hurler Scheie Hurler-Scheie enzyme replacement therapy immune response
On-line přístup:	http://www.sciencedirect.com/science/article/pii/S2329050124002213
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