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Functional correction by antisense therapy of a splicing mutation in the GALT gene
In recent years, antisense therapy has emerged as an increasingly important therapeutic approach to tackle several genetic disorders, including inborn errors of metabolism. Intronic mutations activating cryptic splice sites are particularly amenable to antisense therapy, as the canonical splice site...
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| Publicado no: | Eur J Hum Genet |
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| Main Authors: | , , , , , , , , , |
| Formato: | Artigo |
| Idioma: | Inglês |
| Publicado em: |
Nature Publishing Group
2015
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| Assuntos: | |
| Acesso em linha: | https://ncbi.nlm.nih.gov/pmc/articles/PMC4666580/ https://ncbi.nlm.nih.gov/pubmed/25052314 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/ejhg.2014.149 |
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