Systemic modified messenger RNA for replacement therapy in alpha 1-antitrypsin deficiency

Alpha 1-antitrypsin (AAT) deficiency arises from an inherited mutation in the SERPINA1 gene. The disease causes damage in the liver where the majority of the AAT protein is produced. Lack of functioning circulating AAT protein also causes uninhibited elastolytic activity in the lungs leading to AAT...

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Detalles Bibliográficos
Autores principales: Karadagi, Ahmad, Cavedon, Alex G., Zemack, Helen, Nowak, Greg, Eybye, Marianne E., Zhu, Xuling, Guadagnin, Eleonora, White, Rebecca A., Rice, Lisa M., Frassetto, Andrea L., Strom, Stephen, Jorns, Carl, Martini, Paolo G. V., Ellis, Ewa
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Nature Publishing Group UK 2020
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7184591/
https://www.ncbi.nlm.nih.gov/pubmed/32341402
http://dx.doi.org/10.1038/s41598-020-64017-0

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