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...

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Detalles Bibliográficos
Autores principales: Ioannou, Adam, Fontana, Marianna, Gillmore, Julian D.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Springer International Publishing 2023
Materias:
Review Article
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

Internet

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

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