lipid nanoparticle encapsulating mRNA encoding Cas9 protein and TTR-targeting single guide RNA NTLA-2001

{
  "id": "MLC-T-ONC-014805",
  "term": "lipid nanoparticle encapsulating mRNA encoding Cas9 protein and TTR-targeting single guide RNA NTLA-2001",
  "field": "Oncology and biomedicine",
  "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.",
  "synonyms": [
    "LNP encapsulating mRNA encoding Cas9 protein and TTR-targeting single guide RNA NTLA-2001"
  ],
  "references": [
    "National Cancer Institute Thesaurus"
  ],
  "url": "https://mlchart.com/terminology/oncology/lipid-nanoparticle-encapsulating-mrna-encoding-cas9-protein-and-ttr-targeting/"
}