miR-143 Regulates Lysosomal Enzyme Transport across the Blood-Brain Barrier and Transforms CNS Treatment for Mucopolysaccharidosis Type I

During brain maturation, cation-independent mannose-6-phosphate receptor (CI-MPR), a key transporter for lysosomal hydrolases, decreases significantly on the blood-brain barrier (BBB). Such a phenomenon leads to poor brain penetration of therapeutic enzymes and subsequent failure in reversing neurol...

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Vydáno v:Mol Ther
Hlavní autoři: Lin, Yi, Wang, Xiaohong, Rose, Kevin P., Dai, Mei, Han, Jingfen, Xin, Mei, Pan, Dao
Médium: Artigo
Jazyk:Inglês
Vydáno: American Society of Gene & Cell Therapy 2020
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Original Article
On-line přístup:https://ncbi.nlm.nih.gov/pmc/articles/PMC7544978/
https://ncbi.nlm.nih.gov/pubmed/32610100
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ymthe.2020.06.011
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