Title:
THERAPEUTIC CRISPR/CAS9 GENE EDITING APPROACHES TO THE C9ORF72 REPEAT EXPANSION MUTATION IN IPSCS
Document Type and Number:
WIPO Patent Application WO/2023/220740
Kind Code:
A3
Abstract:
There are provided in vitro and in vivo methods of editing the C9ORF72 repeat expansion mutation using a nuclease to edit a nucleic acid in which the expansion is found. An exemplary method uses a Cas-9 editing system. Guide nucleic acids for editing the repeat expansion mutation are provided. Also provided is a method of mitigating or eliminating symptoms arising in a subject due to the presence of the mutation in the subject's genome.
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Inventors:
CLELLAND CLAIRE (US)
CONKLIN BRUCE (US)
CONKLIN BRUCE (US)
Application Number:
PCT/US2023/066966
Publication Date:
March 21, 2024
Filing Date:
May 12, 2023
Export Citation:
Assignee:
UNIV CALIFORNIA (US)
THE J DAVID GLADSTONE INST A TESTAMENTARY TRUST (US)
THE J DAVID GLADSTONE INST A TESTAMENTARY TRUST (US)
International Classes:
A61K48/00; A61K38/46; A61P25/02; C12N15/113
Domestic Patent References:
| WO2017109757A1 | 2017-06-29 |
Foreign References:
| US20150267197A1 | 2015-09-24 |
Attorney, Agent or Firm:
MANN, Jeffry, S. et al. (US)
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