2019
DOI: 10.1016/j.ymthe.2019.02.019
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Recent Developments in mRNA-Based Protein Supplementation Therapy to Target Lung Diseases

Abstract: The lung has a very unique architecture to enable efficient transfer of oxygen and carbon dioxide required for oxidative metabolism. Inhaled gases travel through the airway tubes via trachea bronchi and bronchioles to the alveoli enriched with blood vessels, the primary site of gas exchange. Inflation and deflation of the lung is a prerequisite for gas exchange at the alveoli. This process requires multiple components like the extracellular matrix, smooth muscle cells, and cartilage for support and flexible co… Show more

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Cited by 75 publications
(70 citation statements)
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References 327 publications
(351 reference statements)
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“…We observed that both chemically modified or unmodified mRNA were accumulated in high abundance in two separate donors compare to the control samples. The mRNA entering the cell in this procedure make the whole blood assay a viable technique to check TLR (3,7 and 8), RIG‐1, and MDA5 mediated immune responses . However, we did not clearly understand the mechanism of reduced DsRed protein expression despite of the presence of magnitude of mRNA transcripts in whole blood cells.…”
Section: Discussionmentioning
confidence: 59%
“…We observed that both chemically modified or unmodified mRNA were accumulated in high abundance in two separate donors compare to the control samples. The mRNA entering the cell in this procedure make the whole blood assay a viable technique to check TLR (3,7 and 8), RIG‐1, and MDA5 mediated immune responses . However, we did not clearly understand the mechanism of reduced DsRed protein expression despite of the presence of magnitude of mRNA transcripts in whole blood cells.…”
Section: Discussionmentioning
confidence: 59%
“…Establishing safe, e cient and reproducible nebulized IVT-mRNA formulations will become the basis of successful gene therapy for various lung based and respiratory diseases, such as cystic brosis and AATD. However, challenges remain in optimizing IVT-mRNA delivery to the lung [19,20], and there are yet very few studies focusing on the nebulized IVT-mRNA formulations designed for AATD treatment. As a result, we set out to investigate optimal conditions for the preparation of A1AT-mRNA/Lipofectamine2000 complexes that can produce su cient A1AT after the nebulization process.…”
Section: Discussionmentioning
confidence: 99%
“…mRNA expression of hACE2 removes these barriers allowing for hACE2 expression in multiple species using a single construct. Additionally, mRNAs can be targeted to specific organs including the liver and lungs [ 49 , 50 ], allowing for localized expression of hACE2 to study the impact of SARS-CoV-2 on specific organs. Finally, as we and others have shown, the mRNA transfection is poorly immunogenic, so repeated administration of the same or different constructs can be administered to the same animal over a prolonged period of time.…”
Section: Discussionmentioning
confidence: 99%