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RNA Targeting and Gene Editing Strategies for Transthyretin Amyloidosis
Transthyretin (TTR) is a tetrameric protein synthesized primarily by the liver. TTR can misfold into pathogenic ATTR amyloid fibrils that deposit in the nerves and heart, causing a progressive and debilitating polyneuropathy (PN) and life-threatening cardiomyopathy (CM). Therapeutic strategies, whic...
| Autores principales: | , , |
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| Formato: | Online Artículo Texto |
| Lenguaje: | English |
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Springer International Publishing
2023
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| Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9933836/ https://www.ncbi.nlm.nih.gov/pubmed/36795354 http://dx.doi.org/10.1007/s40259-023-00577-7 |