Crispr/cas-based base editing composition for restoring dystrophin function
AssigneeDuke University
InventorsCharles A. Gersbach, Veronica Gough
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Abstract
Disclosed herein are CRISPR/Cas-based base editing compositions and methods for treating Duchenne Muscular Dystrophy by restoring dystrophin function. In an aspect, the disclosure relates to a CRISPR/Cas-based base editing system for altering a RNA splice site encoded in the genomic DMA of a subject. In some embodiments, altering the RNA splice site encoded in the genomic DNA results in exclusion or inclusion of at least one exon sequence in an RNA transcript.
Claims33
A CRISPR/Cas-based base editing system for altering an RNA splice site encoded in the genomic DNA of a subject, the CRISPR/Cas-based base editing system comprising a fusion protein and at least one guide RNA (gRNA), wherein the fusion protein comprises a Cas protein and a base-editing domain.
A CRiSPR/Cas-based base editing system for restoring dystrophin function in a subject, the CRISPR/Cas-based base editing system comprising a fusion protein and at least one guide RNA (gRNA), wherein the fusion protein comprises a Cas protein and a base-editing domain.
The CRISPR/Cas-based base editing system of any one of claims 1-18, wherein the base-editing domain comprises one UGI domain or two UGI domains. 20, The CRISPR/Cas-based base editing system of any one of claims 1-19, wherein the fusion protein comprises the structure:
- NH2-[cytidine deaminase domain]-[Cas protein]-[UGI domain]-COOH, and wherein each instance of “-” comprises an optional linker.
Dependencies are inferred from the claim text.