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 <i>IDS</i> 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 lead...
| Main Authors: | , |
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| Format: | Article |
| Language: | English |
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MDPI AG
2021-05-01
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| Series: | International Journal of Molecular Sciences |
| Subjects: | |
| Online Access: | https://www.mdpi.com/1422-0067/22/11/5490 |