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- NCIT_C116792 IAO_0000115 "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." @default.
- NCIT_C116792 NCIT_NHC0 "C116792" @default.
- NCIT_C116792 NCIT_P106 "Pharmacologic Substance" @default.
- NCIT_C116792 NCIT_P108 "Patisiran" @default.
- NCIT_C116792 NCIT_P207 "C3896714" @default.
- NCIT_C116792 NCIT_P210 "1420706-45-1" @default.
- NCIT_C116792 NCIT_P319 "50FKX8CB2Y" @default.
- NCIT_C116792 NCIT_P322 "FDA" @default.
- NCIT_C116792 NCIT_P329 "762821" @default.
- NCIT_C116792 NCIT_P330 "762821" @default.
- NCIT_C116792 NCIT_P399 "762821" @default.
- NCIT_C116792 normalizedInformationContent "100" @default.
- NCIT_C116792 referenceCount "1" @default.
- NCIT_C116792 hasExactSynonym "ALN-18328" @default.
- NCIT_C116792 hasExactSynonym "ALN-TTR02" @default.
- NCIT_C116792 hasExactSynonym "PATISIRAN" @default.
- NCIT_C116792 hasExactSynonym "Patisiran" @default.
- NCIT_C116792 hasExactSynonym "RNAi Therapeutic-targeting Transthyretin ALN-TTR02" @default.
- NCIT_C116792 inSubset NCIT_C176424 @default.
- NCIT_C116792 inSubset NCIT_C63923 @default.
- NCIT_C116792 type Class @default.
- NCIT_C116792 isDefinedBy ncit.owl @default.
- NCIT_C116792 label "Patisiran" @default.
- NCIT_C116792 subClassOf NCIT_C116792 @default.
- NCIT_C116792 subClassOf NCIT_C129821 @default.
- NCIT_C116792 subClassOf NCIT_C1908 @default.
- NCIT_C116792 subClassOf NCIT_C1909 @default.
- NCIT_C116792 subClassOf NCIT_C274 @default.
- NCIT_C116792 subClassOf NCIT_C307 @default.