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Rare Genetic Disorders: Novel Treatment Strategies and Insights Into Human Biology
The last decade has seen a dramatic increase in innovative ideas for the treatment of genetic disorders for which no curative therapies exist. Gene and protein replacement therapies stand out as novel approaches to treat a select group of these diseases, such as certain tissue fragility disorders. F...
| Autores principales: | , |
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| Formato: | Online Artículo Texto |
| Lenguaje: | English |
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Frontiers Media S.A.
2021
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| Materias: | |
| Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8378213/ https://www.ncbi.nlm.nih.gov/pubmed/34422015 http://dx.doi.org/10.3389/fgene.2021.714764 |