Gene therapy for alpha 1-antitrypsin deficiency with an oxidant-resistant human alpha 1-antitrypsin

Alpha 1-antitrypsin (AAT) deficiency, a hereditary disorder characterized by low serum levels of functional AAT, is associated with early development of panacinar emphysema. AAT inhibits serine proteases, including neutrophil elastase, protecting the lung from proteolytic destruction. Cigarette smok...

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Detalles Bibliográficos
Autores principales: Sosulski, Meredith L., Stiles, Katie M., Frenk, Esther Z., Hart, Fiona M., Matsumura, Yuki, De, Bishnu P., Kaminsky, Stephen M., Crystal, Ronald G.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society for Clinical Investigation 2020
Materias:
Research Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7455074/
https://www.ncbi.nlm.nih.gov/pubmed/32759494
http://dx.doi.org/10.1172/jci.insight.135951

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