NCIt definition : A lipid nanoparticle (LNP)-based formulation consisting of small-interfering RNAs
(siRNAs) directed against transthyretin (TTR) encapsulated in lipids, which has potential
use in the treatment of TTR-mediated amyloidosis (ATTR). Upon intravenous administration
of patisiran, the lipid formulation promotes the uptake by cells. The siRNAs bind
to TTR mRNAs, which may result in the inhibition of both the translation and expression
of the TTR protein. ATTR is caused by mutations in the TTR gene and results in the
formation of abnormal amyloid proteins that accumulate in and cause damage to various
body organs and tissues.;