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AssigneesThe Broad Institute, Inc., University of Washington, President and Fellows of Harvard College
InventorsDavid R. Liu, Beverly Mok, Joseph D. Mougous, Snow Brook Peterson, Marcos de Moraes, Julian Willis
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The specification provides programmable base editors that are capable of introducing a nucleotide change and/or which could alter or modify the nucleotide sequence at a target site in mitochondrial DNA (mtDNA) with high specificity and efficiency. Moreover, the disclosure provides fusion proteins and compositions comprising a programmable DNA binding protein (e.g., a mitoTALE, a mitoZFP, or a CRISPR/Casp) and double-stranded DNA deaminase that is capable of being delivered to the mitochondria and carrying out precise installation of nucleotide changes in the mtDNA. The fusion proteins and compositions are not limited for use with mtDNA, but also may be used for base editing of any double-stranded target DNA.
A base editor comprising a heterodimer having first and second monomers, said first monomer comprising a first programmable DNA binding protein and an N-terminal fragment of a split double-stranded DNA deaminase, and said second monomer comprising a second programmable DNA binding protein and a C-terminal fragment of a split double-stranded DNA deaminase,
Dependencies are inferred from the claim text.