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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...

पूर्ण विवरण

में बचाया:
ग्रंथसूची विवरण
में प्रकाशित:Mol Ther
मुख्य लेखकों: Garnacho, Carmen, Dhami, Rajwinder, Solomon, Melani, Schuchman, Edward H., Muro, Silvia
स्वरूप: Artigo
भाषा:Inglês
प्रकाशित: American Society of Gene & Cell Therapy 2017
विषय:
ऑनलाइन पहुंच: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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