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THERAPEUTIC CRISPR/CAS9 GENE EDITING APPROACHES TO THE C9ORF72 REPEAT EXPANSION MUTATION IN IPSCS

Publication EP4709861A2 Kind: A2 Mar 18, 2026

Applicants

THE REGENTS OF THE UNIVERSITY OF CALIFORNIA

Inventors

CLELLAND, Claire

IPC Classifications

C12N 15/113 20100101AFI20251212BHEP C12N 9/22 20060101ALI20251212BHEP A61K 31/7105 20060101ALI20251212BHEP A61K 48/00 20060101ALI20251212BHEP C12N 15/10 20060101ALI20251212BHEP C12N 15/864 20060101ALI20251212BHEP A61P 25/28 20060101ALI20251212BHEP

Designated States

AL, AT, BE, BG, CH, CY, CZ, DE, DK, EE, ES, FI, FR, GB, GR, HR, HU, IE, IS, IT, LI, LT, LU, LV, MC, ME, MK, MT, NL, NO, PL, PT, RO, RS, SE, SI, SK, SM, TR