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In Vitro Investigations on Optimizing and Nebulization of IVT-mRNA Formulations for Potential Pulmonary-Based Alpha-1-Antitrypsin Deficiency Treatment †

In vitro-transcribed (IVT) mRNA has come into focus in recent years as a potential therapeutic approach for the treatment of genetic diseases. The nebulized formulations of IVT-mRNA-encoding alpha-1-antitrypsin (A1AT-mRNA) would be a highly acceptable and tolerable remedy for the protein replacement...

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Detalles Bibliográficos
Autores principales: Guan, Shan, Darmstädter, Max, Xu, Chuanfei, Rosenecker, Joseph
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2021
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8399093/
https://www.ncbi.nlm.nih.gov/pubmed/34452241
http://dx.doi.org/10.3390/pharmaceutics13081281