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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Detalhes bibliográficos
Publicado no: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.
Formato: Artigo
Idioma:Inglês
Publicado em: John Wiley and Sons Inc. 2020
Assuntos:
Original Research Articles
Acesso em linha: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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