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Comment
. 2022 Jul 6;7(1):213.
doi: 10.1038/s41392-022-01068-x.

Prime time for base editing in the mitochondria

Michael A Morgan  1   2 , Lucas Lange  3   4 , Axel Schambach  5   6   7
Affiliations
Comment

Prime time for base editing in the mitochondria

Michael A Morgan et al. Signal Transduct Target Ther. .
No abstract available

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Conflict of interest statement

The authors declare no competing interests.

Figures

Fig. 1
Fig. 1
Challenges and promises of mitochondrial DNA (mtDNA) base editing applications to treat mitochondrial diseases. Efficiency of mtDNA base editing is dependent upon several factors, including efficient delivery to diseased cells, targeting of the mitochondria and mtDNA within these cells, as well as lack of off-target base editing activities. Abbreviations: LHON Leber hereditary optic neuropathy; MELAS mitochondrial encephalopathy, lactic acidosis and stroke-like episodes; DEAF deafness, sensorineural hearing loss; HCM hypertrophic cardiomyopathy; MERFF myoclonus epilepsy associated with ragged red fibers; KSS Kearns-Sayre syndrome; AAV adeno-associated virus; TALENs transcription activator-like effector nucleases; ZFN zinc-finger nucleases; CRISPR clustered regularly interspaced short palindromic repeats; TALEDs transcription activator-like effector deaminases; DdCBE DddA-derived cytosine base editor; MTS mitochondrial targeting sequence; DddAtox double-stranded DNA deaminase toxin A; TadA8e deoxyadenosine deaminase variant
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References

    1. Cho S-I, et al. Targeted A-to-G base editing in human mitochondrial DNA with programmable deaminases. Cell. 2022;185:1764–1776.e12. doi: 10.1016/j.cell.2022.03.039. - DOI - PubMed
    1. Gammage PA, et al. Genome editing in mitochondria corrects a pathogenic mtDNA mutation in vivo. Nat. Med. 2018;24:1691–1695. doi: 10.1038/s41591-018-0165-9. - DOI - PMC - PubMed
    1. Reddy P, et al. Selective elimination of mitochondrial mutations in the germline by genome editing. Cell. 2015;161:459–469. doi: 10.1016/j.cell.2015.03.051. - DOI - PMC - PubMed
    1. Zekonyte U, et al. Mitochondrial targeted meganuclease as a platform to eliminate mutant mtDNA in vivo. Nat. Commun. 2021;12:1–11. doi: 10.1038/s41467-021-23561-7. - DOI - PMC - PubMed
    1. Silva-Pinheiro P, et al. In vivo mitochondrial base editing via adeno-associated viral delivery to mouse post-mitotic tissue. Nat. Commun. 2022;13:1–9. doi: 10.1038/s41467-022-28358-w. - DOI - PMC - PubMed

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