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Exploiting epigenetics for the treatment of inborn errors of metabolism
Gene therapy is currently considered as the optimal treatment for inborn errors of metabolism (IEMs), as it aims to permanently compensate for the primary genetic defect. However, emerging gene editing approaches such as CRISPR‐Cas9, in which the DNA of the host organism is edited at a precise locat...
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| Publicat a: | J Inherit Metab Dis |
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| Autors principals: | , , |
| Format: | Artigo |
| Idioma: | Inglês |
| Publicat: |
John Wiley & Sons, Inc.
2019
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| Matèries: | |
| Accés en línia: | https://ncbi.nlm.nih.gov/pmc/articles/PMC7041640/ https://ncbi.nlm.nih.gov/pubmed/30916397 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1002/jimd.12093 |
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