Enhanced Delivery and Effects of Acid Sphingomyelinase by ICAM-1-Targeted Nanocarriers in Type B Niemann-Pick Disease Mice

Acid sphingomyelinase deficiency in type B Niemann-Pick disease leads to lysosomal sphingomyelin storage, principally affecting lungs, liver, and spleen. Infused recombinant enzyme is beneficial, yet its delivery to the lungs is limited and requires higher dosing than liver and spleen, leading to po...

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Bibliografische gegevens
Gepubliceerd in:Mol Ther
Hoofdauteurs: Garnacho, Carmen, Dhami, Rajwinder, Solomon, Melani, Schuchman, Edward H., Muro, Silvia
Formaat: Artigo
Taal:Inglês
Gepubliceerd in: American Society of Gene & Cell Therapy 2017
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Original Article
Online toegang:https://ncbi.nlm.nih.gov/pmc/articles/PMC5498833/
https://ncbi.nlm.nih.gov/pubmed/28606376
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ymthe.2017.05.014
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