Methods for Treatment of Alport Syndrome
    36.
    发明公开

    公开(公告)号:US20240115598A1

    公开(公告)日:2024-04-11

    申请号:US18446743

    申请日:2023-08-09

    申请人: Sanofi

    发明人: Timothy Wright

    摘要: Provided herein are methods for the treatment of Alport syndrome, using a modified oligonucleotide targeted to miR-21. In certain embodiments, the modified oligonucleotide targeted to miR-21 improves kidney function and/or reduces fibrosis in subjects having Alport syndrome. In certain embodiments, administration of the modified oligonucleotide targeted to miR-21 delays the onset of end-stage renal disease in a subject having Alport syndrome. In certain embodiments, the modified oligonucleotide targeted to miR-21 delays the need for dialysis or kidney transplant in a subject having Alport syndrome.

    STABLE TARGET-EDITING GUIDE RNA TO WHICH CHEMICALLY MODIFIED NUCLEIC ACID IS INTRODUCED

    公开(公告)号:US20240093227A1

    公开(公告)日:2024-03-21

    申请号:US18265756

    申请日:2021-12-08

    IPC分类号: C12N15/85 C12N9/78 C12N15/11

    摘要: Provided is an oligonucleotide which may induce an editing activity of ADRC in cell and has excellent stability in a living body. The oligonucleotide includes a first oligonucleotide identifying a target RNA and a second oligonucleotide linked to the 5′-side of the first oligonucleotide. The first oligonucleotide consists of a target-corresponding nucleotide residue, an oligonucleotide of 10 to 24 residues at the 3′-side, and an oligonucleotide of 3 to 6 residues at the 5′-side. The second oligonucleotide has no nucleotide residue corresponding to a nucleotide residue of the target RNA or has a nucleotide residue which does not form a complementary pair at the 3′-end thereof and the number of residue is 3 to 6. The residue at the 3′-side of the target-corresponding nucleotide residue is a 2′-deoxynucleotide residue, and the third nucleotide residue counted in the 3′-direction from the target-corresponding nucleotide in the oligonucleotide at the 3′-side of the target-corresponding nucleotide residue is a 2′-deoxy-2′-fluoronucleotide residue.