In vivo mitochondrial base editing via adeno-associated viral delivery to mouse post-mitotic tissue

[1]  M. Minczuk,et al.  The potential of mitochondrial genome engineering , 2021, Nature Reviews Genetics.

[2]  Bin Shen,et al.  Precision modeling of mitochondrial diseases in zebrafish via DdCBE-mediated mtDNA base editing , 2021, Cell discovery.

[3]  C. Moraes,et al.  Mitochondrial targeted meganuclease as a platform to eliminate mutant mtDNA in vivo , 2021, Nature Communications.

[4]  S. Karthikeyan,et al.  An atlas of mitochondrial DNA genotype-phenotype associations in the UK Biobank , 2021, Nature Genetics.

[5]  Beum-Chang Kang,et al.  Mitochondrial DNA editing in mice with DddA-TALE fusion deaminases , 2021, Nature Communications.

[6]  Thomas M. Keane,et al.  Twelve years of SAMtools and BCFtools , 2020, GigaScience.

[7]  N. Larsson,et al.  Mitochondrial DNA copy number in human disease: the more the better? , 2020, FEBS letters.

[8]  Apekshya Panda,et al.  MitoCarta3.0: an updated mitochondrial proteome now with sub-organelle localization and pathway annotations , 2020, Nucleic Acids Res..

[9]  J. Stewart Current progress with mammalian models of mitochondrial DNA disease , 2020, Journal of inherited metabolic disease.

[10]  P. Chinnery,et al.  Extreme heterogeneity of human mitochondrial DNA from organelles to populations , 2020, Nature reviews. Genetics.

[11]  David R. Liu,et al.  A bacterial cytidine deaminase toxin enables CRISPR-free mitochondrial base editing , 2020, Nature.

[12]  Tiziano Flati,et al.  HPC-REDItools: a novel HPC-aware tool for improved large scale RNA-editing analysis , 2020, BMC Bioinformatics.

[13]  D. Turnbull,et al.  Mitochondrial Diseases: Hope for the Future , 2020, Cell.

[14]  B. Habermann,et al.  Mitochondrial fusion is required for regulation of mitochondrial DNA replication , 2019, PLoS genetics.

[15]  C. Moraes,et al.  MitoTALEN reduces mutant mtDNA load and restores tRNAAla levels in a mouse model of heteroplasmic mtDNA mutation , 2018, Nature Medicine.

[16]  Pedro Rebelo-Guiomar,et al.  Genome editing in mitochondria corrects a pathogenic mtDNA mutation in vivo , 2018, Nature Medicine.

[17]  S. Rawson,et al.  CryoEM structures of complex I from mouse heart mitochondria in two biochemically-defined states , 2018, Nature Structural & Molecular Biology.

[18]  Ping Liu,et al.  Targeted elimination of mutant mitochondrial DNA in MELAS-iPSCs by mitoTALENs , 2018, Protein & Cell.

[19]  M. Minczuk,et al.  Mitochondrial Genome Engineering: The Revolution May Not Be CRISPR-Ized , 2017, Trends in genetics : TIG.

[20]  A. Suomalainen,et al.  Mitochondrial diseases: the contribution of organelle stress responses to pathology , 2017, Nature Reviews Molecular Cell Biology.

[21]  J. Hirst,et al.  Structure of the Deactive State of Mammalian Respiratory Complex I , 2017, bioRxiv.

[22]  N. Larsson,et al.  Mammalian Mitochondria and Aging: An Update. , 2017, Cell metabolism.

[23]  Laura C. Greaves,et al.  A Phenotype-Driven Approach to Generate Mouse Models with Pathogenic mtDNA Mutations Causing Mitochondrial Disease , 2016, Cell reports.

[24]  Pedro Rebelo-Guiomar,et al.  Near-complete elimination of mutant mtDNA by iterative or dynamic dose-controlled treatment with mtZFNs , 2016, Nucleic acids research.

[25]  Marni J. Falk,et al.  MitoTALEN: A General Approach to Reduce Mutant mtDNA Loads and Restore Oxidative Phosphorylation Function in Mitochondrial Diseases. , 2015, Molecular therapy : the journal of the American Society of Gene Therapy.

[26]  Robert W. Taylor,et al.  Prevalence of nuclear and mitochondrial DNA mutations related to adult mitochondrial disease , 2015, Annals of neurology.

[27]  M. Minczuk,et al.  Mitochondrially targeted ZFNs for selective degradation of pathogenic mitochondrial genomes bearing large-scale deletions or point mutations , 2014, EMBO molecular medicine.

[28]  C. Moraes,et al.  Specific elimination of mutant mitochondrial genomes in patient–derived cells by mitoTALENs , 2013, Nature Medicine.

[29]  Linda Partridge,et al.  Cardioprotection by S-nitrosation of a cysteine switch on mitochondrial complex I , 2013, Nature Medicine.

[30]  Vamsi K. Mootha,et al.  Mitochondrial disorders as windows into an ancient organelle , 2012, Nature.

[31]  Steven L Salzberg,et al.  Fast gapped-read alignment with Bowtie 2 , 2012, Nature Methods.

[32]  S. Dimauro,et al.  Mitochondrial Diseases , 2012, Journal of epilepsy research.

[33]  Christopher A. Miller,et al.  VarScan 2: somatic mutation and copy number alteration discovery in cancer by exome sequencing. , 2012, Genome research.

[34]  D. Duan,et al.  Manipulation of mtDNA heteroplasmy in all striated muscles of newborn mice by AAV9-mediated delivery of a mitochondria targeted restriction endonuclease , 2011, Gene Therapy.

[35]  A. Papa Hope for the future. , 2011, Journal of emergency nursing: JEN : official publication of the Emergency Department Nurses Association.

[36]  Marcel Martin Cutadapt removes adapter sequences from high-throughput sequencing reads , 2011 .

[37]  Paulina Kolasinska-Zwierz,et al.  Construction and testing of engineered zinc-finger proteins for sequence-specific modification of mtDNA , 2010, Nature Protocols.

[38]  Aaron Klug,et al.  Development of a single-chain, quasi-dimeric zinc-finger nuclease for the selective degradation of mutated human mitochondrial DNA , 2008, Nucleic acids research.

[39]  E. Schon,et al.  Selective elimination of mutant mitochondrial genomes as therapeutic strategy for the treatment of NARP and MILS syndromes , 2006, Gene Therapy.

[40]  E. Shoubridge,et al.  Rapid directional shift of mitochondrial DNA heteroplasmy in animal tissues by a mitochondrially targeted restriction endonuclease. , 2005, Proceedings of the National Academy of Sciences of the United States of America.

[41]  S. Srivastava,et al.  Manipulating mitochondrial DNA heteroplasmy by a mitochondrially targeted restriction endonuclease. , 2001, Human molecular genetics.

[42]  N. Gross,et al.  Apparent turnover of mitochondrial deoxyribonucleic acid and mitochondrial phospholipids in the tissues of the rat. , 1969, The Journal of biological chemistry.

[43]  Y. Akao,et al.  Gene therapy for mitochondrial disease by delivering restriction endonuclease SmaI into mitochondria. , 2002, Journal of biomedical science.

[44]  K. Johnson An Update. , 1984, Journal of food protection.