SERPINA1 mRNA as a Treatment for Alpha-1 Antitrypsin Deficiency

Alpha-1-antitrypsin (AAT) deficiency is a genetic disorder that produces inactive/defective AAT due to mutations in the SERPINA1 gene encoding AAT. This disease is associated with decreased activity of AAT in the lungs and deposition of excessive defective AAT protein in the liver. Currently there i...

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Bibliographic Details
Main Authors: Brendan Connolly, Cleo Isaacs, Lei Cheng, Kirtika H. Asrani, Romesh R. Subramanian
Format: Article
Language:English
Published: Wiley 2018-01-01
Series:Journal of Nucleic Acids
Online Access:http://dx.doi.org/10.1155/2018/8247935
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