Related references
Note: Only part of the references are listed.Determining the Minimally Effective Dose of a Clinical Candidate Adeno-Associated Virus Vector in a Mouse Model of Hemophilia A
Jenny A. Greig et al.
HUMAN GENE THERAPY (2022)
Targeting the hepatitis B cccDNA with a sequence-specific ARCUS nuclease to eliminate hepatitis B virus in vivo
Cassandra L. Gorsuch et al.
MOLECULAR THERAPY (2022)
Mitochondrial base editor induces substantial nuclear off-target mutations
Zhixin Lei et al.
NATURE (2022)
Mitochondrial genome engineering coming-of-age
Jose Domingo Barrera-Paez et al.
TRENDS IN GENETICS (2022)
Mitochondrial base editor DdCBE causes substantial DNA off-target editing in nuclear genome of embryos
Yinghui Wei et al.
CELL DISCOVERY (2022)
Onasemnogene abeparvovec for presymptomatic infants with two copies of SMN2 at risk for spinal muscular atrophy type 1: the Phase III SPR1NT trial
Kevin A. Strauss et al.
NATURE MEDICINE (2022)
Modulating Mitochondrial DNA Heteroplasmy with Mitochondrially Targeted Endonucleases
Nikita Mikhailov et al.
ANNALS OF BIOMEDICAL ENGINEERING (2022)
Precise and simultaneous quantification of mitochondrial DNA heteroplasmy and copy number by digital PCR
Wendy K. Shoop et al.
JOURNAL OF BIOLOGICAL CHEMISTRY (2022)
Long-term stable reduction of low-density lipoprotein in nonhuman primates following in vivo genome editing of PCSK9
Lili Wang et al.
MOLECULAR THERAPY (2021)
Tune instead of destroy: How proteolysis keeps OXPHOS in shape
Karolina Szczepanowska et al.
BIOCHIMICA ET BIOPHYSICA ACTA-BIOENERGETICS (2021)
Mitochondrial targeted meganuclease as a platform to eliminate mutant mtDNA in vivo
Ugne Zekonyte et al.
NATURE COMMUNICATIONS (2021)
Strategies for mitochondrial gene editing
Xingbo Yang et al.
COMPUTATIONAL AND STRUCTURAL BIOTECHNOLOGY JOURNAL (2021)
DNA-editing enzymes as potential treatments for heteroplasmic mtDNA diseases
U. Zekonyte et al.
JOURNAL OF INTERNAL MEDICINE (2020)
Therapeutic Manipulation of mtDNA Heteroplasmy: A Shifting Perspective
Christopher B. Jackson et al.
TRENDS IN MOLECULAR MEDICINE (2020)
Organoid cultures of MELAS neural cells reveal hyperactive Notch signaling that impacts neurodevelopment
Winanto et al.
CELL DEATH & DISEASE (2020)
mitoTev-TALE: a monomeric DNA editing enzyme toreduce mutant mitochondrial DNA levels
Claudia V. Pereira et al.
EMBO MOLECULAR MEDICINE (2018)
Meganuclease targeting of PCSK9 in macaque liver leads to stable reduction in serum cholesterol
Lili Wang et al.
NATURE BIOTECHNOLOGY (2018)
Linear mitochondrial DNA is rapidly degraded by components of the replication machinery
Viktoriya Peeva et al.
NATURE COMMUNICATIONS (2018)
The mitochondrial DNA polymerase gamma degrades linear DNA fragments precluding the formation of deletions
Nadee Nissanka et al.
NATURE COMMUNICATIONS (2018)
Targeted elimination of mutant mitochondrial DNA in MELAS-iPSCs by mitoTALENs
Yi Yang et al.
PROTEIN & CELL (2018)
MitoTALEN reduces mutant mtDNA load and restores tRNAAla levels in a mouse model of heteroplasmic mtDNA mutation
Sandra R. Bacman et al.
NATURE MEDICINE (2018)
Genome editing in mitochondria corrects a pathogenic mtDNA mutation in vivo
Payam A. Gammage et al.
NATURE MEDICINE (2018)
Integration of a CD19 CAR into the. TCR Alpha Chain Locus Streamlines Production of Allogeneic Gene-Edited CAR T Cells
Daniel T. MacLeod et al.
MOLECULAR THERAPY (2017)
Single-Dose Gene-Replacement Therapy for Spinal Muscular Atrophy
J. R. Mendell et al.
NEW ENGLAND JOURNAL OF MEDICINE (2017)
TALEN-mediated shift of mitochondrial DNA heteroplasmy in MELAS-iPSCs with m.13513G>A mutation
Naoki Yahata et al.
SCIENTIFIC REPORTS (2017)
Selective mitochondrial DNA degradation following double-strand breaks
Amandine Moretton et al.
PLOS ONE (2017)
Circumventing the Crabtree Effect: A method to induce lactate consumption and increase oxidative phosphorylation in cell culture
Alexandra I. Mot et al.
INTERNATIONAL JOURNAL OF BIOCHEMISTRY & CELL BIOLOGY (2016)
Near-complete elimination of mutant mtDNA by iterative or dynamic dose-controlled treatment with mtZFNs
Payam A. Gammage et al.
NUCLEIC ACIDS RESEARCH (2016)
Mitochondrial diseases
Grainne S. Gorman et al.
NATURE REVIEWS DISEASE PRIMERS (2016)
Prevalence of Nuclear and Mitochondrial DNA Mutations Related to Adult Mitochondrial Disease
Grainne S. Gorman et al.
ANNALS OF NEUROLOGY (2015)
Selective Elimination of Mitochondrial Mutations in the Germline by Genome Editing
Pradeep Reddy et al.
CELL (2015)
MELAS syndrome: Clinical manifestations, pathogenesis, and treatment options
Ayman W. El-Hattab et al.
MOLECULAR GENETICS AND METABOLISM (2015)
Improving Single Injection CSF Delivery of AAV9-mediated Gene Therapy for SMA: A Dose-response Study in Mice and Nonhuman Primates
Kathrin Meyer et al.
MOLECULAR THERAPY (2015)
MitoTALEN: A General Approach to Reduce Mutant mtDNA Loads and Restore Oxidative Phosphorylation Function in Mitochondrial Diseases
Masami Hashimoto et al.
MOLECULAR THERAPY (2015)
The dynamics of mitochondrial DNA heteroplasmy: implications for human health and disease
James B. Stewart et al.
NATURE REVIEWS GENETICS (2015)
Mitochondrially targeted ZFNs for selective degradation of pathogenic mitochondrial genomes bearing large-scale deletions or point mutations
Payam A. Gammage et al.
EMBO MOLECULAR MEDICINE (2014)
The impact of Nucleofection® on the activation state of primary human CD4 T cells
Mingce Zhang et al.
JOURNAL OF IMMUNOLOGICAL METHODS (2014)
Specific elimination of mutant mitochondrial genomes in patient-derived cells by mitoTALENs
Sandra R. Bacman et al.
NATURE MEDICINE (2013)
The Maintenance of Mitochondrial DNA Integrity-Critical Analysis and Update
Mikhail Alexeyev et al.
COLD SPRING HARBOR PERSPECTIVES IN BIOLOGY (2013)
MELAS: A nationwide prospective cohort study of 96 patients in Japan
Shuichi Yatsuga et al.
BIOCHIMICA ET BIOPHYSICA ACTA-GENERAL SUBJECTS (2012)
Manipulation of mtDNA heteroplasmy in all striated muscles of newborn mice by AAV9-mediated delivery of a mitochondria-targeted restriction endonuclease
S. R. Bacman et al.
GENE THERAPY (2012)
Non-specific protein-DNA interactions control I-CreI target binding and cleavage
Rafael Molina et al.
NUCLEIC ACIDS RESEARCH (2012)
Systemic scAAV9 variant mediates brain transduction in newborn rhesus macaques
Benjamin Dehay et al.
SCIENTIFIC REPORTS (2012)
Aerobic Glycolysis: Meeting the Metabolic Requirements of Cell Proliferation
Sophia Y. Lunt et al.
ANNUAL REVIEW OF CELL AND DEVELOPMENTAL BIOLOGY, VOL 27 (2011)
The implications of mitochondrial DNA copy number regulation during embryogenesis
Phillippa J. Carling et al.
MITOCHONDRION (2011)
Systemic Gene Delivery in Large Species for Targeting Spinal Cord, Brain, and Peripheral Tissues for Pediatric Disorders
Adam K. Bevan et al.
MOLECULAR THERAPY (2011)
Natural history of MELAS associated with mitochondrial DNA m.3243A>G genotype
P. Kaufmann et al.
NEUROLOGY (2011)
Organ-specific shifts in mtDNA heteroplasmy following systemic delivery of a mitochondria-targeted restriction endonuclease
S. R. Bacman et al.
GENE THERAPY (2010)
Intravenous Administration of Self-complementary AAV9 Enables Transgene Delivery to Adult Motor Neurons
Sandra Duque et al.
MOLECULAR THERAPY (2009)
Intravascular AAV9 preferentially targets neonatal neurons and adult astrocytes
Kevin D. Foust et al.
NATURE BIOTECHNOLOGY (2009)
Selective elimination of mutant mitochondrial genomes as therapeutic strategy for the treatment of NARP and MILS syndromes
M. F. Alexeyev et al.
GENE THERAPY (2008)
A functionally dominant mitochondrial DNA mutation
Sabrina Sacconi et al.
HUMAN MOLECULAR GENETICS (2008)
Circumventing the crabtree effect: Replacing media glucose with galactose increases susceptibility of HepG2 cells to mitochondrial toxicants
Lisa D. Marroquin et al.
TOXICOLOGICAL SCIENCES (2007)
Population prevalence of the MELAS A3243G mutation
Neil Manwaring et al.
MITOCHONDRION (2007)
Pathogenic mitochondrial DNA mutations in protein-coding genes
Lee-Jun C. Wong
MUSCLE & NERVE (2007)
Modulating mtDNA heteroplasmy by mitochondria-targeted restriction endonucleases in a 'differential multiple cleavage-site' model
S. R. Bacman et al.
GENE THERAPY (2007)
Sequence-specific modification of mitochondrial DNA using a chimeric zinc finger methylase
Michal Minczuk et al.
PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA (2006)
Rapid directional shift of mitochondrial DNA heteroplasmy in animal tissues by a mitochondrially targeted restriction endonuclease
MP Bayona-Bafaluy et al.
PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA (2005)
Precise determination of mitochondrial DNA copy number in human skeletal and cardiac muscle by a PCR-based assay: lack of change of copy number with age
FJ Miller et al.
NUCLEIC ACIDS RESEARCH (2003)
Mitochondrial threshold effects
R Rossignol et al.
BIOCHEMICAL JOURNAL (2003)
The NS2 proteins of parvovirus minute virus of mice are required for efficient nuclear egress of progeny virions in mouse cells
V Eichwald et al.
JOURNAL OF VIROLOGY (2002)
Manipulating mitochondrial DNA heteroplasmy by a mitochondrially targeted restriction endonuclease
S Srivastava et al.
HUMAN MOLECULAR GENETICS (2001)
The genetics and pathology of oxidative phosphorylation
J Smeitink et al.
NATURE REVIEWS GENETICS (2001)
Multisite control of the Crabtree effect in ascites hepatoma cells
S Rodríguez-Enríquez et al.
EUROPEAN JOURNAL OF BIOCHEMISTRY (2001)
What regulates mitochondrial DNA copy number in animal cells?
CT Moraes
TRENDS IN GENETICS (2001)
Share Paper
