Compositions and methods for mitochondrial genome editing
AssigneesCity of Hope, The Regents of the University of California
InventorsJohn Burnett, Anh Pham
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Abstract
Compositions and methods for mitochondria genome editing are provided. Also provided are methods for treating mitochondrial disorders by the disclosed compositions.
Claims44
A composition comprising a delivery vehicle and a protein comprising a mitochondrial localization amino acid sequence covalently attached to an RNA-guided DNA endonuclease enzyme, wherein said protein is bound to said delivery vehicle.
A protein comprising a mitochondrial localization amino acid sequence covalently attached to Cpf1 or a Class II CRISPR endonuclease or a Cas9 variant.
A nucleic acid comprising a mitochondrial import sequence and a single-guide RNA (sgRNA) sequence.
Dependencies are inferred from the claim text.