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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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Detalles Bibliográficos
Publicado en:J Nucleic Acids
Main Authors: Connolly, Brendan, Isaacs, Cleo, Cheng, Lei, Asrani, Kirtika H., Subramanian, Romesh R.
Formato: Artigo
Idioma:Inglês
Publicado: Hindawi 2018
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Acceso en liña:https://ncbi.nlm.nih.gov/pmc/articles/PMC6020464/
https://ncbi.nlm.nih.gov/pubmed/30009048
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1155/2018/8247935
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