Less is More: an overview on the use of RNAi as a tool to achieve Substrate Reduction in Mucopolysaccharidoses
Lysosomal storage diseases (LSDs) are a group of genetic disorders caused by dysfunction in enzymes responsible for the intralysosomal degradation of particular compounds. Given their complex nature and the limitations of available therapies, the shift towards the development of combination treatmen...
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| Other Authors: | , |
| Format: | conferenceObject |
| Language: | eng |
| Published: |
2018
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| Subjects: | |
| Online Access: | http://hdl.handle.net/10400.18/5511 |
| Country: | Portugal |
| Oai: | oai:repositorio.insa.pt:10400.18/5511 |