Transcranial pulsed ultrasound facilitates brain uptake of laronidase in enzyme replacement therapy for Mucopolysaccharidosis type I disease

BACKGROUND: Mucopolysaccharidosis type I (MPS I) is a debilitating hereditary disease characterized by alpha-L-iduronidase (IDUA) deficiency and consequent inability to degrade glycosaminoglycans. The pathological accumulation of glycosaminoglycans systemically results in severe mental retardation a...

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Detalles Bibliográficos
Autores principales: Hsu, Yu-Hone, Liu, Ren-Shyan, Lin, Win-Li, Yuh, Yeong-Seng, Lin, Shuan-Pei, Wong, Tai-Tong
Formato: Online Artículo Texto
Lenguaje:English
Publicado: BioMed Central 2017
Materias:
Research
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5465581/
https://www.ncbi.nlm.nih.gov/pubmed/28595620
http://dx.doi.org/10.1186/s13023-017-0649-6

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5465581/
https://www.ncbi.nlm.nih.gov/pubmed/28595620
http://dx.doi.org/10.1186/s13023-017-0649-6

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