ANGIOPOIETIN-LIKE 3 (ANGPTL3) IRNA COMPOSTIONS AND METHODS OF USE THEREOF 3(ANGPTL3)IRNA

ANGIOPOIETIN-LIKE 3 (ANGPTL3) IRNA COMPOSTIONS AND METHODS OF USE THEREOF 3(ANGPTL3)IRNA

The invention relates to a double-stranded ribonucleic acid (dsRNA) agent for inhibiting expression of Angiopoietin-like 3 (ANGPTL3), comprising a sense strand and an antisense strand forming a duplex region, wherein the sense strand comprises at least 15, preferably at least 16 and most preferably...

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Bibliographische Detailangaben
Hauptverfasser: STUART MILSTEIN S, BRIAN BETTENCOURT B, KEVIN FITZGERALD K, MARIA FRANK-KAMENETSKY M, WILLIAM QUERBES W, SVETLANA SHULGA-MORSKAYA S
Format: Patent
Sprache:chi ; eng
Schlagworte:
CHEMISTRY
DERIVATIVES THEREOF
METALLURGY
NUCLEIC ACIDS
NUCLEOSIDES
NUCLEOTIDES
ORGANIC CHEMISTRY
SUGARS
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Beschreibung
Zusammenfassung:The invention relates to a double-stranded ribonucleic acid (dsRNA) agent for inhibiting expression of Angiopoietin-like 3 (ANGPTL3), comprising a sense strand and an antisense strand forming a duplex region, wherein the sense strand comprises at least 15, preferably at least 16 and most preferably at least 17 contiguous nucleotides of the nucleotide sequence 5'-GUCUCAAAAUGGAAGGUUAUA-3' (SEQ ID NO: 1160), wherein the antisense strand comprises at least 15 contiguous nucleotides of the nucleotide sequence 5'-UAUAACCUUCCAUUUUGAGACUU-3' (SEQ ID NO: 1341), wherein at least one of the modified nucleotides is selected from the group consisting of a 2'-O-methyl modified nucleotide, a 2'-fluoro modified nucleotide, and a nucleotide comprising a 5'-phosphorothioate group, and wherein a ligand comprising an N-acetylgalactosamine (GaINAc) is conjugated to at least one strand of the dsRNA agent.