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