The use of a modified U1 snRNA as a therapeutic strategy to correct a 5’ splice-site mutation in Mucopolysaccharidosis IIIC: in vitro steps towards an in vivo approach
Genetic therapy directed towards the correction of RNA missplicing is being investigated not only at basic research level but even in late-stage clinical trials. Many mutations that change the normal splicing pattern and lead to aberrant mRNA production have been identified in Lysosomal Storage Diso...
| Autor principal: | |
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| Outros Autores: | , , , , |
| Formato: | conferenceObject |
| Idioma: | eng |
| Publicado em: |
2020
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| Assuntos: | |
| Texto completo: | http://hdl.handle.net/10400.18/6792 |
| País: | Portugal |
| Oai: | oai:repositorio.insa.pt:10400.18/6792 |