간외 전달

• Main Authors: MAIER MARTIN, MILSTEIN STUART, KEL'IN ALEXANDER V, FITZGERALD KEVIN, CASTORENO ADAM, PARMAR RUBINA G, CHARISSE KLAUS, NAIR JAYAPRAKASH K, JADHAV VASANT, THEILE CHRISTOPHER S, BROWN KIRK, RAJEEV KALLANTHOTTATHIL G, MANOHARAN MUTHIAH, JAYARAMAN MUTHUSAMY
• Format: Patent
• Language: Korean
• Published: 17.02.2021
• Subjects: BEER; BIOCHEMISTRY; CHEMISTRY; COMPOSITIONS THEREOF; CULTURE MEDIA; ENZYMOLOGY; HUMAN NECESSITIES; HYGIENE; MEDICAL OR VETERINARY SCIENCE; METALLURGY; MICROBIOLOGY; MICROORGANISMS OR ENZYMES; MUTATION OR GENETIC ENGINEERING; PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES; PROPAGATING, PRESERVING OR MAINTAINING MICROORGANISMS; SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS ORMEDICINAL PREPARATIONS; SPIRITS; VINEGAR; WINE

본 발명은 유전자 사일런싱의 방법으로서, 선택적으로 링커 또는 담체를 통해, 적어도 하나의 가닥의 하나 이상의 내부 위치에서 치료적 유효량의 친유성 모이어티-접합 이중-가닥 iRNA를 이를 필요로 하는 세포 또는 피험자에 투여하는 단계를 포함하는, 방법에 관한 것이다. One aspect of the present invention relates to a double stranded iRNA agent comprising an antisense strand which is complementary to a target gene; a sense strand which is complementary to said antisense strand; and one or more lipophilic moieties conjugated to one or more internal positions on at least one strand, optionally via a linker or carrier. Another aspect of the invention relates to a method of gene silencing, comprising administering to a cell or a subject in need thereof a therapeutically effective amount of the lipophilic moieties-conjugated double-stranded iRNAs.