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Gene Therapy for Methylmalonic Acidemia: Past, Present, and Future
Methylmalonic acidemia (MMA) is a severe, and sometimes lethal, monogenic metabolic disorder in need of improved treatments. A number of new genomic therapies, which include canonical adeno-associated virus gene addition, genome editing, and systemic mRNA therapy, have shown great promise in murine...
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| Publicado no: | Hum Gene Ther |
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| Main Authors: | , |
| Formato: | Artigo |
| Idioma: | Inglês |
| Publicado em: |
Mary Ann Liebert, Inc., publishers
2019
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| Assuntos: | |
| Acesso em linha: | https://ncbi.nlm.nih.gov/pmc/articles/PMC6763959/ https://ncbi.nlm.nih.gov/pubmed/31303064 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1089/hum.2019.113 |
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