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Challenges and Prospects for Alpha-1 Antitrypsin Deficiency Gene Therapy

Alpha-1 antitrypsin (AAT) is a protease inhibitor belonging to the serpin family. A number of identified mutations in the SERPINA1 gene encoding this protein result in alpha-1 antitrypsin deficiency (AATD). A decrease in AAT serum concentration or reduced biological activity causes considerable risk...

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Foilsithe in:Hum Gene Ther
Main Authors: Wozniak, Joanna, Wandtke, Tomasz, Kopinski, Piotr, Chorostowska-Wynimko, Joanna
Formáid: Artigo
Teanga:Inglês
Foilsithe: Mary Ann Liebert, Inc. 2015
Ábhair:
Rochtain Ar Líne:https://ncbi.nlm.nih.gov/pmc/articles/PMC4651033/
https://ncbi.nlm.nih.gov/pubmed/26413996
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1089/hum.2015.044
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