Preferred Label : Lipid Nanoparticle Encapsulating mRNA Encoding Cas9 Protein and TTR-targeting Single
Guide RNA NTLA-2001;
NCIt synonyms : LNP Encapsulating mRNA Encoding Cas9 Protein and TTR-targeting Single Guide RNA NTLA-2001;
NCIt definition : A lipid nanoparticle (LNP) formulation encapsulating messenger RNA (mRNA) encoding
the clustered regularly interspaced short palindromic repeat (CRISPR)-associated protein
9 (Cas9) protein and a single guide RNA targeting transthyretin (TTR), that may decrease
serum TTR concentration. Upon administration of LNP encapsulating mRNA encoding Cas9
protein and TTR-targeting single guide RNA NTLA-2001, the LNP moiety binds to the
plasma membrane of nearby cells and releases the Cas9 mRNA and the TTR-targeting single
guide RNA into the cells. The mRNA is then translated by the cellular protein translation
machinery to produce Cas9, which gene-edits the TTR gene in vivo, thereby lowering
the levels of misfolded TTR in the blood. This may reduce the accumulation of misfolded
TTR protein in the tissues.;
Molecule name : NTLA 2001; NTLA-2001;
NCI Metathesaurus CUI : CL1927385;
Origin ID : C204942;
concept_is_in_subset
