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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
Autores principales: Connolly, Brendan, Isaacs, Cleo, Cheng, Lei, Asrani, Kirtika H., Subramanian, Romesh R.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Hindawi 2018
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6020464/
https://www.ncbi.nlm.nih.gov/pubmed/30009048
http://dx.doi.org/10.1155/2018/8247935