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

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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Bibliographic Details
Main Authors: Shan Guan, Max Darmstädter, Chuanfei Xu, Joseph Rosenecker
Format: Article
Language:English
Published: MDPI AG 2021-08-01
Series:Pharmaceutics
Subjects:
gene therapy
IVT-mRNA
alpha-1-antitrypsin deficiency
nebulization
pulmonary delivery
Online Access:https://www.mdpi.com/1999-4923/13/8/1281

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https://www.mdpi.com/1999-4923/13/8/1281

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