CareLit · DOI-Nachweis · Zeitschrift für Gastroenterologie
Hepatocyte-like cell platforms for in vitro evaluation of antisense drugs in familial amyloidosis using stem cell technology
DOI 10.1055/s-0035-1567997
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Autor:innenC Niemietz, V Sauer, J Stella, G Chandhok, A Zibert, HHJ Schmidt
ZeitschriftZeitschrift für Gastroenterologie
Ausgabe12/2015
Jahrgang / SeitenNicht angegeben
Kernaussage aus der Literatur
Abstract-Vorschau
Familial amyloidosis (ATTR) is a neurodegenerative disease caused by mutations of the transthyretin (TTR) gene that is mostly (> 95%) expressed by liver. Currently, more than 100 TTR mutations have been discovered in patients with ATTR, with different clinical manifestation. Drug therapy of ATTR is partially possible on a protein level. However, a full silencing of the mutated TTR gene would enormously increase ther…
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Zitierempfehlung
C Niemietz, V Sauer, J Stella, G Chandhok, A Zibert, HHJ Schmidt (2015) Hepatocyte-like cell platforms for in vitro evaluation of antisense drugs in familial amyloidosis using stem cell technology. Zeitschrift für Gastroenterologie (12). https://doi.org/10.1055/s-0035-1567997
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