Single base editing of atnx2 for the treatment of sca2
AssigneeThe Children's Hospital of Philadelphia
InventorsBeverly DAVIDSON, Defne AMADO, Michael KUCKYR
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Abstract
Provided herein are compositions and methods for inhibiting expression of Ataxin 2 (ATXN2) in target cell comprising subjecting said cell to single base editing that introduces at least one premature stop codon within the coding region for repeat region of ATXN2, thereby terminating translation.
Claims80
A method of inhibiting expression of Ataxin 2 (ATXN2) in a target cell, the method comprising subjecting said cell to single base editing that introduces a premature stop codon within the coding region for the repeat region of ATXN2, thereby terminating translation.
A guide RNA (gRNA) comprising the sequence 5′-GCAGCAGCAGCCGCCGCCCG-3′ (SEQ ID NO:
- 1).
A cell comprising the composition of 57.
Dependencies are inferred from the claim text.