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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...

Täydet tiedot

Tallennettuna:
Bibliografiset tiedot
Julkaisussa:J Nucleic Acids
Päätekijät: Connolly, Brendan, Isaacs, Cleo, Cheng, Lei, Asrani, Kirtika H., Subramanian, Romesh R.
Aineistotyyppi: Artigo
Kieli:Inglês
Julkaistu: Hindawi 2018
Aiheet:
Linkit: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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