COMPOSITIONS AND METHODS FOR MODULATING HBV AND TTR EXPRESSION

• Main Authors: SETH PUNIT P, PRAKASH THAZHA P, SWAYZE ERIC E
• Format: Patent
• Language: English; Japanese
• Published: 29.07.2021
• Subjects: BEER; BIOCHEMISTRY; CHEMISTRY; COMPOSITIONS THEREOF; CULTURE MEDIA; DERIVATIVES THEREOF; ENZYMOLOGY; HUMAN NECESSITIES; HYGIENE; MEDICAL OR VETERINARY SCIENCE; METALLURGY; MICROBIOLOGY; MICROORGANISMS OR ENZYMES; MUTATION OR GENETIC ENGINEERING; NUCLEIC ACIDS; NUCLEOSIDES; NUCLEOTIDES; ORGANIC CHEMISTRY; PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES; PROPAGATING, PRESERVING OR MAINTAINING MICROORGANISMS; SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS ORMEDICINAL PREPARATIONS; SPIRITS; SUGARS; VINEGAR; WINE

To provide conjugated antisense compounds comprising an antisense oligonucleotide complementary to a nucleic acid transcript.SOLUTION: The present invention provides a compound comprising a modified oligonucleotide and a conjugate group, where the modified oligonucleotide consists of 8 to 80 linked nucleosides and has a nucleobase sequence at least 85%, 90%, 95%, or 100% complementary to a sequence encoding transthyretin.SELECTED DRAWING: None 【課題】核酸転写物に相補的なアンチセンスオリゴヌクレオチドを含む共役アンチセンス化合物を提供する。【解決手段】修飾オリゴヌクレオチドおよびコンジュゲート基を含む化合物であって、修飾オリゴヌクレオチドが、８〜８０個の連結したヌクレオシドからなり、かつトランスサイレチンをコードする配列に少なくとも８５％、９０％、９５％、または１００％相補的な核酸塩基配列を有する、化合物である。【選択図】なし