SERPINA1 mRNA as a Treatment for Alpha-1 Antitrypsin Deficiency

Alpha-1-antitrypsin (AAT) deficiency is a genetic disorder that produces inactive/defective AAT due to mutations in the SERPINA1 gene encoding AAT. This disease is associated with decreased activity of AAT in the lungs and deposition of excessive defective AAT protein in the liver. Currently there i...

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Bibliografiska uppgifter
I publikationen:J Nucleic Acids
Huvudupphovsmän: Connolly, Brendan, Isaacs, Cleo, Cheng, Lei, Asrani, Kirtika H., Subramanian, Romesh R.
Materialtyp: Artigo
Språk:Inglês
Publicerad: Hindawi 2018
Ämnen:
Research Article
Länkar:https://ncbi.nlm.nih.gov/pmc/articles/PMC6020464/
https://ncbi.nlm.nih.gov/pubmed/30009048
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1155/2018/8247935
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