Ligand conjugated antisense oligonucleotide for the treatment of transthyretin amyloidosis: preclinical and phase 1 data

AIMS: Amyloidogenic transthyretin (ATTR) amyloidosis is a fatal disease characterized by progressive cardiomyopathy and/or polyneuropathy. AKCEA‐TTR‐L(Rx) (ION‐682884) is a ligand‐conjugated antisense drug designed for receptor‐mediated uptake by hepatocytes, the primary source of circulating transt...

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Published in:ESC Heart Fail
Main Authors: Viney, Nicholas J., Guo, Shuling, Tai, Li‐Jung, Baker, Brenda F., Aghajan, Mariam, Jung, Shiangtung W., Yu, Rosie Z., Booten, Sheri, Murray, Heather, Machemer, Todd, Burel, Sebastien, Murray, Sue, Buchele, Gustavo, Tsimikas, Sotirios, Schneider, Eugene, Geary, Richard S., Benson, Merrill D., Monia, Brett P.
Format: Artigo
Language:Inglês
Published: John Wiley and Sons Inc. 2020
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Original Research Articles
Online Access:https://ncbi.nlm.nih.gov/pmc/articles/PMC7835591/
https://ncbi.nlm.nih.gov/pubmed/33283485
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1002/ehf2.13154
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