Title:
COMPOSITIONS AND METHODS FOR TREATING TRINUCLEOTIDE REPEAT DISORDERS
Document Type and Number:
WIPO Patent Application WO/2024/044708
Kind Code:
A3
Abstract:
Methods and compositions for reducing expansion of nucleotide repeats in a cell, comprising base editors and a guide RNA (gRNA) that directs the Cas-based enzyme to the splice acceptor site for mutL homolog 3 (MLH3) exon 7 or to the MLH3 endonuclease domain.
Inventors:
PINTO RICARDO (US)
KLEINSTIVER BENJAMIN (US)
KLEINSTIVER BENJAMIN (US)
Application Number:
PCT/US2023/072851
Publication Date:
April 25, 2024
Filing Date:
August 24, 2023
Export Citation:
Assignee:
MASSACHUSETTS GEN HOSPITAL (US)
International Classes:
C12N15/10
Attorney, Agent or Firm:
DEYOUNG, Janice, Kugler et al. (US)
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