Title:
初代細胞において標的核酸の遺伝子調節を誘導するためにインビトロで行う方法
Document Type and Number:
Japanese Patent JP6873911
Kind Code:
B2
Abstract:
Provided herein are methods for inducing CRISPR/Cas-based gene regulation (e.g., genome editing or gene expression) of a target nucleic acid (e.g., target DNA or target RNA) in a cell. The methods include using modified single guide RNAs (sgRNAs) that enhance gene regulation of the target nucleic acid in a primary cell for use in ex vivo therapy or in a cell in a subject for use in in vivo therapy. Additionally, provided herein are methods for preventing or treating a genetic disease in a subject by administering a sufficient amount of a modified sgRNA to correct a mutation in a target gene associated with the genetic disease.
Inventors:
Porteas, Matthew H
Eaal Handel
Clark, joe
Baku, Rasmus Oh
Ryan, Daniel Yee
Dellinger, Douglas Jay
Robert Kaiser
Joel Meyerson
Eaal Handel
Clark, joe
Baku, Rasmus Oh
Ryan, Daniel Yee
Dellinger, Douglas Jay
Robert Kaiser
Joel Meyerson
Application Number:
JP2017552447A
Publication Date:
May 19, 2021
Filing Date:
April 05, 2016
Export Citation:
Assignee:
INTERNATIONAL BUSINESS MACHINES CORPORATION
International Classes:
C12N15/09; C12N5/10
Foreign References:
| WO2014093712A1 | ||||
| WO2014093635A1 | ||||
| WO2015026885A1 |
Other References:
PNAS,2015年11月16日,E7110-E7117
Nature Biotechnol.,2015年 9月,vol.33,p.985-989
Nature Biotechnol.,2015年 9月,vol.33,p.985-989
Attorney, Agent or Firm:
Shoichi Okuyama
Arihara Koichi
Matsushima Tetsuo
Ayako Nakamura
Satoshi Morimoto
Yu Tanaka
Tokumoto Koichi
Akiko Mizushima
Arihara Koichi
Matsushima Tetsuo
Ayako Nakamura
Satoshi Morimoto
Yu Tanaka
Tokumoto Koichi
Akiko Mizushima