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Enhancing the Therapeutic Potential of Sulfamidase for the Treatment of Mucopolysaccharidosis IIIA
Mucopolysaccharidosis type IIIA (MPS-IIIA) is a lysosomal storage disorder (LSD) caused by inherited defect of sulfamidase, a lysosomal sulfatase. MPS-IIIA is one of the most common and severe forms of LSDs with CNS involvement. Presently there is no cure. Here we have developed a new gene delivery...
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| Vydáno v: | Mol Ther Methods Clin Dev |
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| Hlavní autoři: | , , , , , , , , , , , , , , |
| Médium: | Artigo |
| Jazyk: | Inglês |
| Vydáno: |
American Society of Gene & Cell Therapy
2019
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| Témata: | |
| On-line přístup: | https://ncbi.nlm.nih.gov/pmc/articles/PMC6881609/ https://ncbi.nlm.nih.gov/pubmed/31788497 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.omtm.2019.10.009 |
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