Title:
TEMPLATE-INDEPENDENT GENOME EDITING AND REPAIRING CORRECT FRAME-SHIFTING DISEASE IN VIVO
Document Type and Number:
WIPO Patent Application WO/2021/208937
Kind Code:
A1
Abstract:
Provided is a method for repairing a frame-shift mutation in a gene in a cell. Also provided is a method for treating a disease related to a frame-shift mutation in a subject. These methods can be used to treat diseases associated with animal or human deafness.
Inventors:
XIONG WEI (CN)
SUN QIANWEN (CN)
LIU LIAN (CN)
LI KUAN (CN)
SUN QIANWEN (CN)
LIU LIAN (CN)
LI KUAN (CN)
Application Number:
PCT/CN2021/087119
Publication Date:
October 21, 2021
Filing Date:
April 14, 2021
Export Citation:
Assignee:
UNIV TSINGHUA (CN)
International Classes:
C12N15/63; A61P27/16
Foreign References:
| CN108823235A | 2018-11-16 | |||
| CN109536527A | 2019-03-29 |
Other References:
JOSE BONAFONT, ÁNGELES MENCíA, MARTA GARCíA, RAúL TORRES, SANDRA RODRíGUEZ, MARTA CARRETERO, ESTEBAN CHAC: "Clinically Relevant Correction of Recessive Dystrophic Epidermolysis Bullosa by Dual sgRNA CRISPR/Cas9-Mediated Gene Editing", MOLECULAR THERAPY, ELSEVIER INC., US, vol. 27, no. 5, 1 May 2019 (2019-05-01), US , pages 986 - 998, XP055664831, ISSN: 1525-0016, DOI: 10.1016/j.ymthe.2019.03.007
Attorney, Agent or Firm:
CHENG & PENG INTELLECTUAL PROPERTY LAW OFFICE (CN)
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