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    • 6. 发明申请
    • NUCLEIC ACID CARRIERS AND THERAPEUTIC METHODS OF USE
    • 核酸载体和治疗方法的使用
    • WO2017143171A1
    • 2017-08-24
    • PCT/US2017/018343
    • 2017-02-17
    • GENISPHERE LLC
    • GETTS, Robert, C.KADUSHIN, James, M.GETTS, Lori
    • A61K47/48A61K49/00A61K49/12C08G83/00
    • A61K47/6881A61K47/6803A61K47/6811A61K47/6849A61K47/6889C08G83/00
    • The present disclosure provides nucleic acid carriers comprising one or more layers comprising multiple monomers, wherein each monomer comprises: a first and a second oligonucleotide; wherein a central portion of each of the first and second oligonucleotides is complementary to each other, forming a double-stranded region, wherein the two terminal portions of the first oligonucleotide are not complementary to the two terminal portions of the second oligonucleotide, forming four terminal arms; and at least one targeting agent conjugated to at least one peripheral terminal arm, wherein when the at least one targeting agent is an miRNA. The present disclosure also provides pharmaceutical composition comprising any of the nucleic acid carriers described herein, and a pharmaceutically acceptable vehicle. The present disclosure also provides methods of inducing an immune response in an animal comprising administering to the animal any of the nucleic acid carriers described herein.
    • 本公开提供了包含一个或多个包含多个单体的层的核酸载体,其中每个单体包含:第一和第二寡核苷酸; 其中第一和第二寡核苷酸各自的中心部分互相互补,形成双链区,其中第一寡核苷酸的两个末端部分不与第二寡核苷酸的两个末端部分互补,形成四个末端 武器; 和至少一种缀合于至少一个外围末端臂的靶向剂,其中当所述至少一种靶向剂是miRNA时。 本公开内容还提供了包含本文所述的任何核酸载体和药学上可接受的载体的药物组合物。 本公开内容还提供了在动物中诱导免疫应答的方法,包括向动物施用本文所述的任何核酸载体。