Title:
INHIBITORY NEURON-SPECIFIC PROMOTER
Document Type and Number:
WIPO Patent Application WO/2023/219140
Kind Code:
A1
Abstract:
An inhibitory neuron-specific promoter sequence is discovered, which has a shorter length compared with a mGAD65 promoter and is not deteriorated in all of a promoter activity, specificity to an inhibitory neuron and a gene expression efficiency. More specifically, a promoter is provided, which comprises DNA having a promoter activity, in which the DNA comprises a nucleotide sequence for a Dlx1 binding site and/or a Dlx2 binding site in a glutamic acid decarboxylase (GAD) promoter and a nucleotide sequence for a region lying between the 5'-terminal of exon 1 and a transcription start point (TSS) in a glutamic acid decarboxylase (GAD).
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Inventors:
HIRAI HIROKAZU (JP)
KONNO AYUMU (JP)
MATSUZAKI YASUNORI (JP)
FUKAI YUKI (JP)
KONNO AYUMU (JP)
MATSUZAKI YASUNORI (JP)
FUKAI YUKI (JP)
Application Number:
PCT/JP2023/017785
Publication Date:
November 16, 2023
Filing Date:
May 11, 2023
Export Citation:
Assignee:
UNIV GUNMA NAT UNIV CORP (JP)
International Classes:
C12N15/864; A61K35/76; A61K48/00; A61P25/00; A61P25/08; A61P25/18; A61P43/00; C12N7/01
Other References:
HOSHINO CHIAKI, KONNO AYUMU, HOSOI NOBUTAKE, KANEKO RYOSUKE, MUKAI RYO, NAKAI JUNICHI, HIRAI HIROKAZU: "GABAergic neuron-specific whole-brain transduction by AAV-PHP.B incorporated with a new GAD65 promoter", MOLECULAR BRAIN, BIOMED CENTRAL LTD, LONDON UK, vol. 14, no. 1, 1 December 2021 (2021-12-01), London UK , XP093108971, ISSN: 1756-6606, DOI: 10.1186/s13041-021-00746-1
HOSHINO CHIAKI, KONNO AYUMU, HOSOI NOBUTAKE, KANEKO RYOSUKE, MUKAI RYO, NAKAI JUNICHI, HIRAI HIROKAZU: "GABAergic neuron-specific whole-brain transduction by AAV-PHP.B incorporated with a new GAD65 promoter", MOLECULAR BRAIN, BIOMED CENTRAL LTD, LONDON UK, vol. 14, no. 1, 1 December 2021 (2021-12-01), London UK , XP093108971, ISSN: 1756-6606, DOI: 10.1186/s13041-021-00746-1
YING CHEN; ERBO DONG; DENNIS R. GRAYSON;: "Analysis of the GAD1 promoter: Trans-acting factors and DNA methylation converge on the 5 untranslated region", NEUROPHARMACOLOGY, ELSEVIER, AMSTERDAM, NL, vol. 60, no. 7, 16 September 2010 (2010-09-16), AMSTERDAM, NL, pages 1075 - 1087, XP028193367, ISSN: 0028-3908, DOI: 10.1016/j.neuropharm.2010.09.017
HOSHINO CHIAKI, KONNO AYUMU, HOSOI NOBUTAKE, KANEKO RYOSUKE, MUKAI RYO, NAKAI JUNICHI, HIRAI HIROKAZU: "GABAergic neuron-specific whole-brain transduction by AAV-PHP.B incorporated with a new GAD65 promoter", MOLECULAR BRAIN, BIOMED CENTRAL LTD, LONDON UK, vol. 14, no. 1, 1 December 2021 (2021-12-01), London UK , XP093108971, ISSN: 1756-6606, DOI: 10.1186/s13041-021-00746-1
YING CHEN; ERBO DONG; DENNIS R. GRAYSON;: "Analysis of the GAD1 promoter: Trans-acting factors and DNA methylation converge on the 5 untranslated region", NEUROPHARMACOLOGY, ELSEVIER, AMSTERDAM, NL, vol. 60, no. 7, 16 September 2010 (2010-09-16), AMSTERDAM, NL, pages 1075 - 1087, XP028193367, ISSN: 0028-3908, DOI: 10.1016/j.neuropharm.2010.09.017
Attorney, Agent or Firm:
IP FIRM SHUWA (JP)
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