Title:
GENOME EDITING SYSTEM FOR REPEAT EXPANSION MUTATION
Document Type and Number:
WIPO Patent Application WO/2018/230976
Kind Code:
A1
Abstract:
The present specification pertains to an artificial manipulation or modification in an expression regulatory gene of an over-amplified repeat sequence. More specifically, the present specification pertains to an expression regulatory system of an over-amplified repeat sequence, comprising an artificially manipulated gene to decrease the expression of an over-amplified repeat sequence. The artificially manipulated gene includes at least one selected from the group consisting of a SPT4 gene, a SPT5 gene, a SUPT4H gene, and a SUPT5H gene. The artificially manipulated gene includes an artificial mutation in the genomic sequence of the gene. The artificially manipulated gene includes a gene which is functionally damaged or has an expression level reduced.
Inventors:
LEE JAE YOUNG (KR)
KIM SEOKJOONG (KR)
BAE HEESOOK (KR)
SONG JIHWAN (KR)
PARK HYUN JUNG (KR)
KIM SEOKJOONG (KR)
BAE HEESOOK (KR)
SONG JIHWAN (KR)
PARK HYUN JUNG (KR)
Application Number:
PCT/KR2018/006731
Publication Date:
December 20, 2018
Filing Date:
June 14, 2018
Export Citation:
Assignee:
TOOLGEN INC (KR)
COLLEGE OF MEDICINE POCHON CHA UNIV INDUSTRY ACADEMIC COOPERATION FOUNDATION (KR)
COLLEGE OF MEDICINE POCHON CHA UNIV INDUSTRY ACADEMIC COOPERATION FOUNDATION (KR)
International Classes:
C12N15/86; A61K48/00; C12N9/22; C12N15/10; C12N15/113; C12N15/90
Domestic Patent References:
| WO2016011080A2 | 2016-01-21 | |||
| WO2016095037A1 | 2016-06-23 |
Foreign References:
| US20170051354A1 | 2017-02-23 | |||
| US20160319261A1 | 2016-11-03 |
Other References:
CHENG, HUI-MIN ET AL.: "Effects on Murine Behavior and Lifespan of Selectively Decreasing Expression of Mutant Huntingtin Allele by Supt4h Knockdown", PLOS GENETICS, vol. 11, no. 3, 2015, pages 1 - 17, XP055334085
JINEK ET AL., SCIENCE, vol. 343, no. 6176, 2014, pages 1247997
HIROSHI NISHIMASU ET AL., CELL, vol. 156, 2014, pages 935 - 949
ANDERS ET AL., NATURE, 2014
TAKASHI YAMANO ET AL., CELL, vol. 165, 2016, pages 949 - 962
LEE ET AL., NANO LETT., vol. 12, 2012, pages 6322 - 6327
"NCBI", Database accession no. NM-009296
See also references of EP 3640334A4
JINEK ET AL., SCIENCE, vol. 343, no. 6176, 2014, pages 1247997
HIROSHI NISHIMASU ET AL., CELL, vol. 156, 2014, pages 935 - 949
ANDERS ET AL., NATURE, 2014
TAKASHI YAMANO ET AL., CELL, vol. 165, 2016, pages 949 - 962
LEE ET AL., NANO LETT., vol. 12, 2012, pages 6322 - 6327
"NCBI", Database accession no. NM-009296
See also references of EP 3640334A4
Attorney, Agent or Firm:
IPS PATENT FIRM (KR)
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