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Evaluation of Therapeutic Oligonucleotides for Familial Amyloid Polyneuropathy in Patient-Derived Hepatocyte-Like Cells

Familial amyloid polyneuropathy (FAP) is caused by mutations of the transthyretin (TTR) gene, predominantly expressed in the liver. Two compounds that knockdown TTR, comprising a small interfering RNA (siRNA; ALN-TTR-02) and an antisense oligonucleotide (ASO; IONIS-TTR(Rx)), are currently being eval...

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Detalles Bibliográficos
Autores principales:	Niemietz, Christoph J., Sauer, Vanessa, Stella, Jacqueline, Fleischhauer, Lutz, Chandhok, Gursimran, Guttmann, Sarah, Avsar, Yesim, Guo, Shuling, Ackermann, Elizabeth J., Gollob, Jared, Monia, Brett P., Zibert, Andree, Schmidt, Hartmut H. -J.
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	Public Library of Science 2016
Materias:	Research Article
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5008816/ https://www.ncbi.nlm.nih.gov/pubmed/27584576 http://dx.doi.org/10.1371/journal.pone.0161455

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