Genetically modulated substrate reduction therapy
The concept of enzyme replacement as a potential therapeutic approach to ameliorate lysosomal storage disorders (LSDs) is virtually as old as the concept of LSD itself. In fact, right after the first enzymatic deficiency underlying a LSD was described, both concepts were established and, for years,...
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| Other Authors: | , , |
| Format: | conferenceObject |
| Language: | eng |
| Published: |
2018
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| Subjects: | |
| Online Access: | http://hdl.handle.net/10400.18/5516 |
| Country: | Portugal |
| Oai: | oai:repositorio.insa.pt:10400.18/5516 |