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September 22, 2005Proceedings of the National Academy of SciencesOpen Access

Rapid directional shift of mitochondrial DNA heteroplasmy in animal tissues by a mitochondrially targeted restriction endonuclease

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Why the study?

Does mitochondrially targeted restriction endonuclease ApaLI shift mtDNA heteroplasmy in heteroplasmic mice?

Population

Heteroplasmic mice and cultured hepatocytes from these mice

Design

Preclinical

Follow-up

2-6 hours (in vitro)

Authors

MBM. Pilar Bayona‐BafaluyUniversidad de ZaragozaBBBas BlitsUniQure (Netherlands)BBBrendan J. BattersbyUniversity of Helsinki

Discussion

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Implication

Hypothesis-generating for mtDNA disease therapy; human trials required before clinical consideration.

Structured PICO

Does mitochondrially targeted restriction endonuclease ApaLI shift mtDNA heteroplasmy in heteroplasmic mice?

P
Population
Heteroplasmic mice (with two mtDNA haplotypes, one containing an ApaLI site) and cultured hepatocytes from these mice
I
Intervention
Mitochondrially targeted restriction endonuclease ApaLI (via transfection in vitro and recombinant viral vectors in vivo)
O
Outcome
Shift in mtDNA heteroplasmysurrogate

Expression of a mitochondrially targeted restriction endonuclease can rapidly and directionally shift mtDNA heteroplasmy in vivo, offering a potential therapeutic strategy for mitochondrial diseases.

Cite This Study

Bayona‐Bafaluy et al. (2005) studied this question.

synapsesocial.com/papers/69d770abf44a16d01ef310dbhttps://doi.org/10.1073/pnas.0502896102
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