Adeno-associated virus vector
    2.
    发明授权

    公开(公告)号:US11845952B2

    公开(公告)日:2023-12-19

    申请号:US16968397

    申请日:2019-01-16

    摘要: One disadvantage of adeno-associated virus (AAV) is low gene expression efficiency due to delayed expression of the inserted gene. Provided is a vector plasmid for producing a vector genome to be inserted into an adeno-associated virus (AAV). The vector plasmid is provided with a vector genome cassette as a template of the vector genome, and the vector genome cassette is provided with: (1) an expression cassette including a nucleic acid molecule encoding a target gene and (b) a nucleic acid molecule encoding an expression regulatory region that allows expression of the target gene; and (2) two nucleic acid molecules positioned on both sides of the expression cassette and encoding inverted terminal repeats (ITRs). Also provided is a pharmaceutical composition containing the AVV vector, for treating or preventing an eye disease or disease associated with same. Further provided is a method for treating or preventing the eye disease or disease associated with same, comprising administering a therapeutically effective amount of the pharmaceutical composition for treating or preventing the eye disease or disease associated with same to a subject suffering from the eye disease or disease associated with same.