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AAV-vector based gene therapy for mitochondrial disease: progress and future perspectives
Mitochondrial diseases are a group of rare, heterogeneous diseases caused by gene mutations in both nuclear and mitochondrial genomes that result in defects in mitochondrial function. They are responsible for significant morbidity and mortality as they affect multiple organ systems and particularly...
| Autores principales: | , , |
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| Formato: | Online Artículo Texto |
| Lenguaje: | English |
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BioMed Central
2022
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| Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9169410/ https://www.ncbi.nlm.nih.gov/pubmed/35668433 http://dx.doi.org/10.1186/s13023-022-02324-7 |