Tegsedi (Inotersen): An antisense oligonucleotide approved for the treatment of adult patients with hereditary transthyretin amyloidosis

Tegsedi (Inotersen) is a chemicallymodified 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 an...

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Bibliographic Details
Main Author: Gales, L (author)
Format: article
Language:eng
Published: 2019
Subjects:
Antisense oligonucleotide
Gene silencing
HATTR treatment
Hereditary transthyretin-mediated amyloidosis (hATTR)
Tegsedi (Inotersen)
Transthyretin
Online Access:https://hdl.handle.net/10216/136219
Country:Portugal
Oai:oai:repositorio-aberto.up.pt:10216/136219

Internet

https://hdl.handle.net/10216/136219

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