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Tegsedi (Inotersen): An Antisense Oligonucleotide Approved for the Treatment of Adult Patients with Hereditary Transthyretin Amyloidosis

Tegsedi (Inotersen) is a chemically modified antisense oligonucleotide that inhibits the hepatic production of transthyretin (TTR). Several single-point mutations in TTR destabilize its structure, leading to the aggregation and accumulation of amyloid deposits in the nervous system, heart, kidneys a...

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Detaylı Bibliyografya
Yayımlandı:Pharmaceuticals (Basel)
Yazar: Gales, Luís
Materyal Türü: Artigo
Dil:Inglês
Baskı/Yayın Bilgisi: MDPI 2019
Konular:
Online Erişim:https://ncbi.nlm.nih.gov/pmc/articles/PMC6631675/
https://ncbi.nlm.nih.gov/pubmed/31117178
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.3390/ph12020078
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