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Gene therapy in a murine model of Methylmalonic Acidemia (MMA) using rAAV9 mediated gene delivery
Methylmalonic acidemia (MMA), an inherited metabolic disorder caused by deficient activity of methylmalonyl-CoA mutase (MUT), carries a poor prognosis for long-term survival. While administration of a recombinant adeno-associated virus serotype 8 vector (rAAV8) can rescue Mut(-/-) mice from neonatal...
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| Autors principals: | , , , , |
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| Format: | Artigo |
| Idioma: | Inglês |
| Publicat: |
2011
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| Matèries: | |
| Accés en línia: | https://ncbi.nlm.nih.gov/pmc/articles/PMC3382069/ https://ncbi.nlm.nih.gov/pubmed/21776024 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/gt.2011.108 |
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