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Mysteries of α1-antitrypsin deficiency: emerging therapeutic strategies for a challenging disease

The classical form of α1-antitrypsin deficiency (ATD) is an autosomal co-dominant disorder that affects ~1 in 3000 live births and is an important genetic cause of lung and liver disease. The protein affected, α1-antitrypsin (AT), is predominantly derived from the liver and has the function of inhib...

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Autori principali: Ghouse, Raafe, Chu, Andrew, Wang, Yan, Perlmutter, David H.
Natura: Artigo
Lingua:Inglês
Pubblicazione: The Company of Biologists Limited 2014
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Accesso online:https://ncbi.nlm.nih.gov/pmc/articles/PMC3974452/
https://ncbi.nlm.nih.gov/pubmed/24719116
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1242/dmm.014092
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