Φορτώνει......
Differential Uptake of NAGLU-IGF2 and Unmodified NAGLU in Cellular Models of Sanfilippo Syndrome Type B
Sanfilippo syndrome type B, or mucopolysaccharidosis IIIB (MPS IIIB), is a rare autosomal recessive lysosomal storage disease caused by a deficiency of α-N-acetylglucosaminidase (NAGLU). Deficiency in NAGLU disrupts the lysosomal turnover of heparan sulfate (HS), which results in the abnormal accumu...
Αποθηκεύτηκε σε:
| Τόπος έκδοσης: | Mol Ther Methods Clin Dev |
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| Κύριοι συγγραφείς: | , , , , , , , , , , |
| Μορφή: | Artigo |
| Γλώσσα: | Inglês |
| Έκδοση: |
American Society of Gene & Cell Therapy
2019
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| Θέματα: | |
| Διαθέσιμο Online: | https://ncbi.nlm.nih.gov/pmc/articles/PMC6606967/ https://ncbi.nlm.nih.gov/pubmed/31309128 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.omtm.2019.05.008 |
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