Title:
GENE THERAPY USING GENOME EDITING WITH SINGLE AAV VECTOR
Document Type and Number:
WIPO Patent Application WO/2023/120536
Kind Code:
A9
Abstract:
To provide a vector and a method widely applicable to the treatment of various genetic diseases or the correction of many mutations. A method for inserting a desired nucleic acid into a nucleic acid in a cell by cleaving a target nucleic acid sequence at least at one position in the nucleic acid in the cell, said method comprising: a step for introducing a vector containing the desired nucleic acid into the cell, wherein the vector contains at least one gRNA target sequence located on each side of the desired nucleic acid, the desired nucleic acid, a promoter specific to the cell, a sequence encoding a Cas nuclease, a promoter enabling the expression of the gRNA in the cell after the introduction of the vector, and a sequence encoding the gRNA; and a step for disposing the cell under such conditions as to allow the occurrence of the cleavage. The vector is configured to include a cleavage site that enables the Cas nuclease to form a nucleic acid fragment containing the desired nucleic acid.
Inventors:
NISHIGUCHI KOJI (JP)
FUJITA KOSUKE (JP)
FUJITA KOSUKE (JP)
Application Number:
PCT/JP2022/046944
Publication Date:
August 17, 2023
Filing Date:
December 20, 2022
Export Citation:
Assignee:
NATIONAL UNIV CORPORATION TOKAI NATIONAL HIGHER EDUCATION AND RESEARCH SYSTEM (JP)
International Classes:
C12N15/09; A61K35/76; A61K48/00; A61P27/02; C12N15/85; C12N15/864
Attorney, Agent or Firm:
YAMAMOTO, Shusaku et al. (JP)
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