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Genome editing for inborn errors of metabolism: advancing towards the clinic
Inborn errors of metabolism (IEM) include many disorders for which current treatments aim to ameliorate disease manifestations, but are not curative. Advances in the field of genome editing have recently resulted in the in vivo correction of murine models of IEM. Site-specific endonucleases, such as...
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| Publicado no: | BMC Med |
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| Main Authors: | , , , |
| Formato: | Artigo |
| Idioma: | Inglês |
| Publicado em: |
BioMed Central
2017
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| Assuntos: | |
| Acesso em linha: | https://ncbi.nlm.nih.gov/pmc/articles/PMC5327528/ https://ncbi.nlm.nih.gov/pubmed/28238287 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1186/s12916-017-0798-4 |
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