Less is More: an overview on the use of RNAi as a tool to achieve Substrate Reduction in Mucopolysaccharidoses
Mucopolysaccharidoses (MPSs) are a subgroup of Lysosomal Storage Diseases (LSDs) caused by dysfunction in enzymes responsible for the intralysosomal degradation of glycosaminoglycans (GAGs). Given their complex nature and the limitations of available therapies, the shift towards the development of c...
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| Other Authors: | , |
| Format: | conferenceObject |
| Language: | eng |
| Published: |
2018
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| Subjects: | |
| Online Access: | http://hdl.handle.net/10400.18/5509 |
| Country: | Portugal |
| Oai: | oai:repositorio.insa.pt:10400.18/5509 |