STABLE TARGET-EDITING GUIDE RNA HAVING CHEMICALLY MODIFIED NUCLEIC ACID INTRODUCED
THEREINTO

    公开(公告)号:US20230061732A1

    公开(公告)日:2023-03-02

    申请号:US17616430

    申请日:2020-06-04

    摘要: An oligonucleotide that induces a site-specific editing for a target RNA, the oligonucleotide including a first oligonucleotide that specifies the target RNA, a second oligonucleotide that is linked to the 3′-side of the first oligonucleotide, a third oligonucleotide that is capable of forming a complementary pair together with the second oligonucleotide, and a first linking portion that links the second oligonucleotide and the third oligonucleotide. The first oligonucleotide is composed of a target-corresponding nucleotide residue that corresponds to an adenosine residue in the target RNA; an oligonucleotide of 10 to 30 residues linked to the 5′-side of the target-corresponding nucleotide residue and having a base sequence complementary to the target RNA; and an oligonucleotide of 3-6 residues linked to the 3′-side of the target-corresponding nucleotide residue and having a base sequence complementary to the target RNA. The second oligonucleotide consists of 5 to 8 residues and the third oligonucleotide consists of 5 to 8 residues. At least one residue selected from a counter region composed of the target-corresponding nucleotide residue and two respective residues on the 3′- and 5′-sides thereof is a nucleotide residue other than a natural ribonucleotide residue.

    ANTIBODY RECOGNIZING EXTRACELLULAR REGION OF ALK2/ACVR1

    公开(公告)号:US20220267453A1

    公开(公告)日:2022-08-25

    申请号:US17629715

    申请日:2020-07-22

    IPC分类号: C07K16/28

    摘要: This application provides: an antibody or an antigen-binding fragment thereof which can be used not only as a therapeutic drug but as a tool of immunological and immunohistological analysis such as immunostaining or Western blot, specifically recognizes an extracellular region polypeptide of human, mouse and rat ALK2, and inhibits BMP signal transduction, and an antibody or an antigen-binding fragment thereof which has a property of specifically binding to an extracellular region of ALK2 to form an ALK2 homodimer and a property of inhibiting the formation of an ALK2-type II receptor heterodimer, and inhibits the activation of ALK2 kinase, or inhibits BMP signal transduction; and a method for inhibiting the activation of ALK2 kinase or a method for inhibiting BMP signal transduction using the antibody or the antigen-binding fragment thereof.