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Setting the Stage for Replacement of Mitochondrial Genes

Description: Project Narrative We will develop a DNA editing platform to eliminated mutant mtDNA (mitoARCUS) in human and mouse models. In addition, we will test a newly described base editing tool (DdCBE) to edit mitochondrial DNA in a mouse model.
Funding type: research
Funder scheme: R01
Start date: 1994-12-01
End date: 2027-08-31
Investigator: CARLOS TORRES MORAES
Institution: UNIVERSITY OF MIAMI SCHOOL OF MEDICINE
Funded outputs count: 126
Funder award ID: R01EY010804
OpenAlex ID: G9000898187
The power of one: The unexpected benefits of correcting a single tissue in multisystem mitochondrial disease
2026 · Carlos T. Moraes · Molecular Therapy
Dopaminergic neurons preferentially accumulate mtDNA rearrangements
2026 · Tania Arguello, Christian D. Alcalde Pretel, et al. · Proceedings of the National Academy of Sciences
Generation and characterization of the TRE-TNFR2 transgenic mouse for cell-specific and timed (over)expression of tumor necrosis factor receptor 2
2026 · Placido Illiano, Nadee Nissanka, et al. · Cytokine
Mitochondrial NMNAT3 reduces amyloid aggregates in Drosophila and alters the amyloid processing in the 3xTgAD mouse
2026 · Zhu Yi, Naynika Juvvadi, et al. · Mitochondrial Communications · 1
Transient muscle expression of mitoARCUS in mice leads to sustained reductions in pathogenic mtDNA and reduces fatigability
2025 · Sandra R. Bacman, Wendy Shoop, et al. · Molecular Therapy · 2
mtDNA base editing: Mind the gap
2025 · Jose Domingo Barrera-Paez, Carlos T. Moraes · Molecular Cell
Rewriting nuclear epigenetic scripts in mitochondrial diseases as a strategy for heteroplasmy control
2025 · María J Pérez, Rocío B Colombo, et al. · EMBO Molecular Medicine · 2
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Correcting a pathogenic mitochondrial DNA mutation by base editing in mice
2025 · Jose D. Barrera-Paez, Sandra R. Bacman, et al. · Science Translational Medicine · 17
NEK10 kinase ablation affects mitochondrial morphology, function and protein phosphorylation status
2024 · Andressa Peres de Oliveira, Claudia D. C. Navarro, et al. · Proteome Science · 4
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Restoration of defective oxidative phosphorylation to a subset of neurons prevents mitochondrial encephalopathy
2024 · Brittni R Walker, Lise-Michelle Theard, et al. · EMBO Molecular Medicine · 8
Tools for editing the mammalian mitochondrial genome
2024 · Carlos T Moraes · Human Molecular Genetics · 16
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Absence of both MGME1 and POLG EXO abolishes mtDNA whereas absence of either creates unique mtDNA duplications
2024 · Christian D. Gonzalez, Nadee Nissanka, et al. · Journal of Biological Chemistry · 9
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mitoTALEN reduces the mutant mtDNA load in neurons
2024 · Sandra R. Bacman, Jose Domingo Barrera-Paez, et al. · Molecular Therapy — Nucleic Acids · 22
Efficient elimination of MELAS-associated m.3243G mutant mitochondrial DNA by an engineered mitoARCUS nuclease
2023 · Wendy K. Shoop, Janel Lape, et al. · Nature Metabolism · 46
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Precise and simultaneous quantification of mitochondrial DNA heteroplasmy and copy number by digital PCR
2022 · Wendy K. Shoop, Cassandra L. Gorsuch, et al. · Journal of Biological Chemistry · 24
Adult-Onset Deficiency of Mitochondrial Complex III in a Mouse Model of Alzheimer’s Disease Decreases Amyloid Beta Plaque Formation
2022 · Milena Pinto, Francisca Diaz, et al. · Molecular Neurobiology · 5
Mitochondrial genome engineering coming-of-age
2022 · Jose Domingo Barrera-Paez, Carlos T. Moraes · Trends in Genetics · 45
Nuclear-Mitochondrial Interactions
2022 · Brittni R. Walker, Carlos T. Moraes · Biomolecules · 133
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ATAD3A has a scaffolding role regulating mitochondria inner membrane structure and protein assembly
2021 · Tania Arguello, Susana Peralta, et al. · Cell Reports · 88
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Enhanced glycolysis and GSK3 inactivation promote brain metabolic adaptations following neuronal mitochondrial stress
2021 · Sofia Garcia, Amy Saldana-Caboverde, et al. · Human Molecular Genetics · 17
Mitochondrial targeted meganuclease as a platform to eliminate mutant mtDNA in vivo
2021 · Ugne Zekonyte, Sandra R. Bacman, et al. · Nature Communications · 94
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Mild deficiency of mitochondrial Complex III in a mouse model of Alzheimer’s disease decreases amyloid beta plaque formation
2021 · milena pinto, Francisca Diaz, et al. · Research Square
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Metformin delays neurological symptom onset in a mouse model of neuronal complex I deficiency
2020 · Susana Peralta, Milena Pinto, et al. · JCI Insight · 21
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DNA‐editing enzymes as potential treatments for heteroplasmic mtDNA diseases
2020 · U. Zekonyte, S. R. Bacman, et al. · Journal of Internal Medicine · 29
Mitochondrial DNA heteroplasmy in disease and targeted nuclease‐based therapeutic approaches
2020 · Nadee Nissanka, Carlos T Moraes · EMBO Reports · 123
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