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Article

SERPINA1 mRNA as a Treatment for Alpha-1 Antitrypsin Deficiency

13 Jun 2018

Abstract excerpt

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 is no specific treatment for liver disease associated with AAT deficiency. AAT lung disease is often treated with one...

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Identifiers

PMID: 30009048
DOI: 10.1155/2018/8247935

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