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Gene therapy for Mucopolysaccharidoses
Mucopolysaccharidoses (MPS) are a group of lysosomal storage disorders (LSDs) caused by a deficiency of lysosomal enzymes, leading to a wide range of various clinical symptoms depending upon the type of MPS or its severity. Enzyme replacement therapy (ERT), hematopoietic stem cell transplantation (H...
Gorde:
| Argitaratua izan da: | Mol Genet Metab |
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| Egile Nagusiak: | , , , , |
| Formatua: | Artigo |
| Hizkuntza: | Inglês |
| Argitaratua: |
2017
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| Gaiak: | |
| Sarrera elektronikoa: | https://ncbi.nlm.nih.gov/pmc/articles/PMC5986190/ https://ncbi.nlm.nih.gov/pubmed/29295764 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ymgme.2017.12.434 |
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