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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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| Publicado no: | Hum Gene Ther |
|---|---|
| Main Authors: | , , , |
| Formato: | Artigo |
| Idioma: | Inglês |
| Publicado em: |
Mary Ann Liebert, Inc.
2015
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| Assuntos: | |
| Acesso em linha: | 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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