Using antisense technology to develop a novel therapy for α-1 antitrypsin deficient (AATD) liver disease and to model AATD lung disease

Alpha-1 antitrypsin (AAT) is a serum protease inhibitor that belongs to the serpin superfamily. Mutations in AAT are associated with α-1 antitrypsin deficiency (AATD), a rare genetic disease with two distinct manifestations: AATD lung disease and AATD liver disease. AATD lung disease is caused by lo...

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Autors principals: Guo, Shuling, Booten, Sheri L, Watt, Andrew, Alvarado, Luis, Freier, Susan M, Teckman, Jeffery H, McCaleb, Michael L, Monia, Brett P
Format: Artigo
Idioma:Inglês
Publicat: Landes Bioscience 2014
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Accés en línia:https://ncbi.nlm.nih.gov/pmc/articles/PMC4091453/
https://ncbi.nlm.nih.gov/pubmed/25054094
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.4161/rdis.28511
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