Title:
MEDICINE FOR DISEASE CAUSED BY FRAME-SHIFT MUTATION
Document Type and Number:
WIPO Patent Application WO/2023/163131
Kind Code:
A1
Abstract:
The present disclosure provides a medicine for treating or preventing a genetic disease, etc. The present disclosure provides a medicine for altering, preventing, or treating a condition, disease, disorder or symptom, which is caused by a frame-shift mutation due to the insertion or deletion of one or more bases, by cleaving at least one target nucleic acid sequence in a nucleic acid within a cell, said medicine comprising a vector. This vector contains a construct that contains a promoter specific to the cell, a sequence encoding a Cas nuclease, a promoter enabling the expression of a gRNA in the cell after the introduction of the vector, and a sequence encoding a gRNA that binds to the target nucleic acid sequence having the insertion or deletion. This construct is configured so as to form a cleavage site, by the Cas nuclease, in proximity to the target nucleic acid sequence having the insertion or deletion.
Inventors:
NISHIGUCHI KOJI (JP)
FUJITA KOSUKE (JP)
ASHIDA YUHEI (JP)
FUJITA KOSUKE (JP)
ASHIDA YUHEI (JP)
Application Number:
PCT/JP2023/006843
Publication Date:
August 31, 2023
Filing Date:
February 24, 2023
Export Citation:
Assignee:
NATIONAL UNIV CORPORATION TOKAI NATIONAL HIGHER EDUCATION AND RESEARCH SYSTEM (JP)
JAPAN CHEM RES (JP)
JAPAN CHEM RES (JP)
International Classes:
A61K48/00; A61K31/7105; A61K35/76; A61K38/46; A61P27/02; C12N15/09; C12N15/55; C12N15/63; C12N15/864
Domestic Patent References:
| WO2020096049A1 | 2020-05-14 |
Foreign References:
| JP2012139170A | 2012-07-26 |
Other References:
KOO TAEYOUNG, LU-NGUYEN NGOC B., MALERBA ALBERTO, KIM EUNJI, KIM DAESIK, CAPPELLARI ORNELLA, CHO HEE-YEON, DICKSON GEORGE, POPPLEW: "Functional Rescue of Dystrophin Deficiency in Mice Caused by Frameshift Mutations Using Campylobacter jejuni Cas9", MOLECULAR THERAPY, ELSEVIER INC., US, vol. 26, no. 6, 1 June 2018 (2018-06-01), US , pages 1529 - 1538, XP055869416, ISSN: 1525-0016, DOI: 10.1016/j.ymthe.2018.03.018
HOSONO KATSUHIRO, ISHIGAMI CHIE, TAKAHASHI MASAYO, PARK DONG HO, HIRAMI YASUHIKO, NAKANISHI HIROSHI, UENO SHINJI, YOKOI TADASHI, H: "Two Novel Mutations in the EYS Gene Are Possible Major Causes of Autosomal Recessive Retinitis Pigmentosa in the Japanese Population", PLOS ONE, vol. 7, no. 2, 17 February 2012 (2012-02-17), pages e31036, XP093086903, DOI: 10.1371/journal.pone.0031036
HOSONO KATSUHIRO, ISHIGAMI CHIE, TAKAHASHI MASAYO, PARK DONG HO, HIRAMI YASUHIKO, NAKANISHI HIROSHI, UENO SHINJI, YOKOI TADASHI, H: "Two Novel Mutations in the EYS Gene Are Possible Major Causes of Autosomal Recessive Retinitis Pigmentosa in the Japanese Population", PLOS ONE, vol. 7, no. 2, 17 February 2012 (2012-02-17), pages e31036, XP093086903, DOI: 10.1371/journal.pone.0031036
Attorney, Agent or Firm:
YAMAMOTO, Shusaku et al. (JP)
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