Title:
METHOD FOR TREATING MYOPATHIES BY TARGETING TITIN GENE
Document Type and Number:
WIPO Patent Application WO/2023/190935
Kind Code:
A1
Abstract:
The present invention aims to provide a novel therapeutic approach to human myopathy (particularly titin-based myopathy). The present invention provides a polynucleotide comprising the following base sequences: (a) a base sequence encoding a fusion protein of a nuclease-deficient CRISPR effector protein and a transcription activator, and (b) a base sequence encoding a guide RNA targeting a continuous region set forth in SEQ ID NO: 36, 37, 38, 39, 46, 47, 51, 52, 56, 65, 66, 67 or 69 in the putative promoter region of human TTN gene.
Inventors:
FERGUSON DANIEL (US)
YAMAGATA TETSUYA (US)
YAMAGATA TETSUYA (US)
Application Number:
PCT/JP2023/013303
Publication Date:
October 05, 2023
Filing Date:
March 30, 2023
Export Citation:
Assignee:
MODALIS THERAPEUTICS CORP (JP)
International Classes:
C12N15/09; A61K31/7105; A61K35/76; A61K35/761; A61K48/00; A61P21/00; C12N15/62; C12N15/861; C12N15/864; C12N15/867
Domestic Patent References:
| WO2020101042A1 | 2020-05-22 | |||
| WO2017087395A1 | 2017-05-26 |
Other References:
STREZOSKA ŽAKLINA; DICKERSON SARAH MICHELLE; MAKSIMOVA ELENA; CHOU ELDON; GROSS MAREN MAYER; HEMPHILL KEVIN; HARDCASTLE TRAVI: "CRISPR-mediated transcriptional activation with synthetic guide RNA", JOURNAL OF BIOTECHNOLOGY, ELSEVIER, AMSTERDAM NL, vol. 319, 27 May 2020 (2020-05-27), Amsterdam NL , pages 25 - 35, XP086200395, ISSN: 0168-1656, DOI: 10.1016/j.jbiotec.2020.05.005
Attorney, Agent or Firm:
KAMADA, Mitsunori et al. (JP)
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