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Gene Therapy for Mucopolysaccharidosis Type II—A Review of the Current Possibilities
Mucopolysaccharidosis type II (MPS II) is a lysosomal storage disorder based on a mutation in the IDS gene that encodes iduronate 2-sulphatase. As a result, there is an accumulation of glycosaminoglycans—heparan sulphate and dermatan sulphate—in almost all body tissues, which leads to their dysfunct...
| Autores principales: | , |
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| Formato: | Online Artículo Texto |
| Lenguaje: | English |
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MDPI
2021
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| Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8197095/ https://www.ncbi.nlm.nih.gov/pubmed/34070997 http://dx.doi.org/10.3390/ijms22115490 |