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Antisense Mediated Splicing Modulation For Inherited Metabolic Diseases: Challenges for Delivery
In the past few years, research in targeted mutation therapies has experienced significant advances, especially in the field of rare diseases. In particular, the efficacy of antisense therapy for suppression of normal, pathogenic, or cryptic splice sites has been demonstrated in cellular and animal...
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| Main Authors: | , , , |
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| Formato: | Artigo |
| Idioma: | Inglês |
| Publicado em: |
Mary Ann Liebert, Inc.
2014
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| Assuntos: | |
| Acesso em linha: | https://ncbi.nlm.nih.gov/pmc/articles/PMC3922136/ https://ncbi.nlm.nih.gov/pubmed/24506780 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1089/nat.2013.0453 |
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