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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Detalhes bibliográficos
Publicado no:JCI Insight
Main Authors: Sosulski, Meredith L., Stiles, Katie M., Frenk, Esther Z., Hart, Fiona M., Matsumura, Yuki, De, Bishnu P., Kaminsky, Stephen M., Crystal, Ronald G.
Formato: Artigo
Idioma:Inglês
Publicado em: American Society for Clinical Investigation 2020
Assuntos:
Research Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC7455074/
https://ncbi.nlm.nih.gov/pubmed/32759494
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1172/jci.insight.135951
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