(Q37767167)

22 August 2017

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title

Mitochondrial medicine: to a new era of gene therapy for mitochondrial DNA mutations (English)

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22 August 2017

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2

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22 August 2017

Marisol Corral-Debrinski

series ordinal

3

object named as

Marisol Corral-Debrinski

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22 August 2017

Hélène Cwerman-Thibault

series ordinal

1

object named as

Hélène Cwerman-Thibault

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22 August 2017

publication date

23 June 2010

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Journal of Inherited Metabolic Disease

22 August 2017

published in

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22 August 2017

volume

34

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22 August 2017

issue

2

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22 August 2017

page(s)

327-344

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https://scigraph.springernature.com/pub.10.1007/s10545-010-9131-5

22 August 2017

exact match

0 references

cites work

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Success in sight: The eyes have it! Ocular gene therapy trials for LCA look promising

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Effect of gene therapy on visual function in Leber's congenital amaurosis

21 January 2018

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Neuroprotective effect of long-term NDI1 gene expression in a chronic mouse model of Parkinson disorder

21 January 2018

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The variability of the harlequin mouse phenotype resembles that of human mitochondrial-complex I-deficiency syndromes

21 January 2018

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Therapeutic potential of dichloroacetate for pyruvate dehydrogenase complex deficiency.

21 January 2018

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Heart transplantation for progressive cardiomyopathy as a manifestation of MELAS syndrome

21 January 2018

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Heart transplantation in children with mitochondrial cardiomyopathy

21 January 2018

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Allotopic mRNA localization to the mitochondrial surface rescues respiratory chain defects in fibroblasts harboring mitochondrial DNA mutations affecting complex I or v subunits

21 January 2018

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The optimized allotopic expression of ND1 or ND4 genes restores respiratory chain complex I activity in fibroblasts harboring mutations in these genes

21 January 2018

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Systematic identification of human mitochondrial disease genes through integrative genomics

21 January 2018

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Human gene therapy for RPE65 isomerase deficiency activates the retinoid cycle of vision but with slow rod kinetics

21 January 2018

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Human RPE65 gene therapy for Leber congenital amaurosis: persistence of early visual improvements and safety at 1 year

21 January 2018

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Vision 1 year after gene therapy for Leber's congenital amaurosis

21 January 2018

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In yeast, the 3' untranslated region or the presequence of ATM1 is required for the exclusive localization of its mRNA to the vicinity of mitochondria

21 January 2018

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https://api.crossref.org/works/10.1007%2FS10545-010-9131-5

DQAsome-mediated delivery of plasmid DNA toward mitochondria in living cells

21 January 2018

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Expression of the alternative oxidase complements cytochrome c oxidase deficiency in human cells

21 January 2018

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Purification and characterization of a rotenone-insensitive NADH:Q6 oxidoreductase from mitochondria of Saccharomyces cerevisiae

21 January 2018

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https://api.crossref.org/works/10.1007%2FS10545-010-9131-5

Neurological effects of high-dose idebenone in patients with Friedreich's ataxia: a randomised, placebo-controlled trial

21 January 2018

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Mitochondrial diseases: therapeutic approaches

21 January 2018

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Mitochondrial disorders in the nervous system

21 January 2018

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https://api.crossref.org/works/10.1007%2FS10545-010-9131-5

A critical approach to the therapy of mitochondrial respiratory chain and oxidative phosphorylation diseases

21 January 2018

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https://api.crossref.org/works/10.1007%2FS10545-010-9131-5

Abnormal neurological features predict poor survival and should preclude liver transplantation in patients with deoxyguanosine kinase deficiency

21 January 2018

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https://api.crossref.org/works/10.1007%2FS10545-010-9131-5

Dichloroacetate therapy attenuates the blood lactate response to submaximal exercise in patients with defects in mitochondrial energy metabolism

21 January 2018

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https://api.crossref.org/works/10.1007%2FS10545-010-9131-5

Tom20 mediates localization of mRNAs to mitochondria in a translation-dependent manner

21 January 2018

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https://api.crossref.org/works/10.1007%2FS10545-010-9131-5

Optimized allotopic expression of the human mitochondrial ND4 prevents blindness in a rat model of mitochondrial dysfunction

21 January 2018

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https://api.crossref.org/works/10.1007%2FS10545-010-9131-5

A role for mitochondrial enzymes in inherited neoplasia and beyond

21 January 2018

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https://api.crossref.org/works/10.1007%2FS10545-010-9131-5

A mouse model of mitochondrial disease reveals germline selection against severe mtDNA mutations

21 January 2018

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https://api.crossref.org/works/10.1007%2FS10545-010-9131-5

Proteome characterization of mouse brain mitochondria using electrospray ionization tandem mass spectrometry

21 January 2018

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https://api.crossref.org/works/10.1007%2FS10545-010-9131-5

Thalamic metabolism and symptom onset in preclinical Huntington's disease

21 January 2018

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https://api.crossref.org/works/10.1007%2FS10545-010-9131-5

Expression of the Ciona intestinalis alternative oxidase (AOX) in Drosophila complements defects in mitochondrial oxidative phosphorylation

21 January 2018

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https://api.crossref.org/works/10.1007%2FS10545-010-9131-5

Mitochondrial presequence and open reading frame mediate asymmetric localization of messenger RNA

21 January 2018

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https://api.crossref.org/works/10.1007%2FS10545-010-9131-5

Coenzyme Q(10) and idebenone in the therapy of respiratory chain diseases: rationale and comparative benefits

21 January 2018

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https://api.crossref.org/works/10.1007%2FS10545-010-9131-5

Rescue of a mitochondrial deficiency causing Leber Hereditary Optic Neuropathy

21 January 2018

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https://api.crossref.org/works/10.1007%2FS10545-010-9131-5

Efficiency and safety of AAV-mediated gene delivery of the human ND4 complex I subunit in the mouse visual system

21 January 2018

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https://api.crossref.org/works/10.1007%2FS10545-010-9131-5

Allotopic expression of a mitochondrial alternative oxidase confers cyanide resistance to human cell respiration

21 January 2018

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https://api.crossref.org/works/10.1007%2FS10545-010-9131-5

How can we treat mitochondrial encephalomyopathies? Approaches to therapy

21 January 2018

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Safety and tolerability of gene therapy with an adeno-associated virus (AAV) borne GAD gene for Parkinson's disease: an open label, phase I trial

21 January 2018

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Cochlear implantation in patients with MELAS syndrome

21 January 2018

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https://api.crossref.org/works/10.1007%2FS10545-010-9131-5

Generation of trans-mitochondrial mice carrying homoplasmic mtDNAs with a missense mutation in a structural gene using ES cells.

21 January 2018

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https://api.crossref.org/works/10.1007%2FS10545-010-9131-5

Dichloroacetate causes toxic neuropathy in MELAS: a randomized, controlled clinical trial

21 January 2018

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Treatment of mitochondrial electron transport chain disorders: a review of clinical trials over the past decade

21 January 2018

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https://api.crossref.org/works/10.1007%2FS10545-010-9131-5

Approaches to finding the molecular basis of mitochondrial oxidative phosphorylation disorders

21 January 2018

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https://api.crossref.org/works/10.1007%2FS10545-010-9131-5

The harlequin mouse mutation downregulates apoptosis-inducing factor

21 January 2018

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https://api.crossref.org/works/10.1007%2FS10545-010-9131-5

Mitochondrial fragmentation in neurodegeneration

21 January 2018

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https://api.crossref.org/works/10.1007%2FS10545-010-9131-5

Mitochondrial medicine: entering the era of treatment

21 January 2018

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https://api.crossref.org/works/10.1007%2FS10545-010-9131-5

Nuclear DNA-encoded tRNAs targeted into mitochondria can rescue a mitochondrial DNA mutation associated with the MERRF syndrome in cultured human cells.

21 January 2018

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https://api.crossref.org/works/10.1007%2FS10545-010-9131-5

Plant mitochondria actively import DNA via the permeability transition pore complex

21 January 2018

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https://api.crossref.org/works/10.1007%2FS10545-010-9131-5

Progress and prospects: gene therapy for mitochondrial DNA disease

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS10545-010-9131-5

OPA1 functions in mitochondria and dysfunctions in optic nerve.

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS10545-010-9131-5

Mitochondrial dynamics in mammalian health and disease

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS10545-010-9131-5

Function and biogenesis of iron-sulphur proteins

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS10545-010-9131-5

Mitochondrial protein import and human health and disease

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS10545-010-9131-5

Safety and efficacy of gene transfer for Leber's congenital amaurosis

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS10545-010-9131-5

Age-dependent effects of RPE65 gene therapy for Leber's congenital amaurosis: a phase 1 dose-escalation trial

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS10545-010-9131-5

Rescue of a deficiency in ATP synthesis by transfer of MTATP6, a mitochondrial DNA-encoded gene, to the nucleus

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS10545-010-9131-5

Genome-wide analysis of mRNAs targeted to yeast mitochondria

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS10545-010-9131-5

Protection by the NDI1 gene against neurodegeneration in a rotenone rat model of Parkinson's disease

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS10545-010-9131-5

In Saccharomyces cerevisiae, ATP2 mRNA sorting to the vicinity of mitochondria is essential for respiratory function

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS10545-010-9131-5

Import of nuclear deoxyribonucleic acid coded lysine-accepting transfer ribonucleic acid (anticodon C-U-U) into yeast mitochondria

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS10545-010-9131-5

Mitochondrial disease--its impact, etiology, and pathology.

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS10545-010-9131-5

A co-translational model to explain the in vivo import of proteins into HeLa cell mitochondria

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS10545-010-9131-5

Targeting peptide nucleic acid (PNA) oligomers to mitochondria within cells by conjugation to lipophilic cations: implications for mitochondrial DNA replication, expression and disease

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS10545-010-9131-5

Assembly of functional proton-translocating ATPase complex in yeast mitochondria with cytoplasmically synthesized subunit 8, a polypeptide normally encoded within the organelle

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS10545-010-9131-5

Complete loss-of-function of the heart/muscle-specific adenine nucleotide translocator is associated with mitochondrial myopathy and cardiomyopathy

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS10545-010-9131-5

Yeast NDI1 improves oxidative phosphorylation capacity and increases protection against oxidative stress and cell death in cells carrying a Leber's hereditary optic neuropathy mutation

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS10545-010-9131-5

The mutant human ND4 subunit of complex I induces optic neuropathy in the mouse

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS10545-010-9131-5

The use of lentiviral vectors to obtain transgenic rats

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS10545-010-9131-5

Synthesis of trifunctional PNA-benzophenone derivatives for mitochondrial targeting, selective DNA binding, and photo-cross-linking

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS10545-010-9131-5

Yeast mitochondrial biogenesis: a role for the PUF RNA-binding protein Puf3p in mRNA localization

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS10545-010-9131-5

Infantile encephalomyopathy and nephropathy with CoQ10 deficiency: a CoQ10-responsive condition.

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS10545-010-9131-5

Generation of transgenic non-human primates with germline transmission

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS10545-010-9131-5

Prevalence of mitochondrial DNA disease in adults

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS10545-010-9131-5

Clinical experience with high-dose idebenone in Friedreich ataxia

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS10545-010-9131-5

Gene therapy for Leber's congenital amaurosis is safe and effective through 1.5 years after vector administration

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS10545-010-9131-5

A review of cochlear implantation in mitochondrial sensorineural hearing loss

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS10545-010-9131-5

Prospects for retinal gene replacement therapy.

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS10545-010-9131-5

Strategies for treating disorders of the mitochondrial genome

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS10545-010-9131-5

Double-strand breaks of mouse muscle mtDNA promote large deletions similar to multiple mtDNA deletions in humans

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS10545-010-9131-5

Controlled clinical trial of dichloroacetate for treatment of congenital lactic acidosis in children

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS10545-010-9131-5

Role of dichloroacetate in the treatment of genetic mitochondrial diseases

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS10545-010-9131-5

Versatility of AAV vectors for retinal gene transfer

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS10545-010-9131-5

The role of the 3' untranslated region in mRNA sorting to the vicinity of mitochondria is conserved from yeast to human cells

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS10545-010-9131-5

Long mRNAs coding for yeast mitochondrial proteins of prokaryotic origin preferentially localize to the vicinity of mitochondria

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS10545-010-9131-5

Selective inhibition of mutant human mitochondrial DNA replication in vitro by peptide nucleic acids

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS10545-010-9131-5

In-vitro genetic modification of mitochondrial function

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS10545-010-9131-5

Somatic mtDNA mutations cause aging phenotypes without affecting reactive oxygen species production

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS10545-010-9131-5

AIF deficiency compromises oxidative phosphorylation

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS10545-010-9131-5

Coenzyme Q-responsive Leigh's encephalopathy in two sisters

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS10545-010-9131-5

The pathophysiology of mitochondrial disease as modeled in the mouse

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS10545-010-9131-5

The role of mitochondria in apoptosis*.

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS10545-010-9131-5

The role of abnormal mitochondrial dynamics in the pathogenesis of Alzheimer's disease

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS10545-010-9131-5

Treatment of late infantile neuronal ceroid lipofuscinosis by CNS administration of a serotype 2 adeno-associated virus expressing CLN2 cDNA.

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS10545-010-9131-5

Mitochondrial drug delivery systems for macromolecule and their therapeutic application to mitochondrial diseases

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS10545-010-9131-5

Caspase-independent death of Leber's hereditary optic neuropathy cybrids is driven by energetic failure and mediated by AIF and Endonuclease G.

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS10545-010-9131-5

OPA1 mutations associated with dominant optic atrophy impair oxidative phosphorylation and mitochondrial fusion

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS10545-010-9131-5

Progressive loss of mitochondrial DNA in thymidine kinase 2-deficient mice

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS10545-010-9131-5

Mitochondrial ABC proteins in health and disease

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS10545-010-9131-5

Apoptosis-Inducing Factor Deficiency Induces Early Mitochondrial Degeneration in Brain Followed by Progressive Multifocal Neuropathology

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS10545-010-9131-5

MITO-Porter: A liposome-based carrier system for delivery of macromolecules into mitochondria via membrane fusion

21 January 2018

1 reference

12 December 2020

Efficient expression of self-complementary AAV in ganglion cells of the ex vivo primate retina

1 reference

12 December 2020

Expression of algal nuclear ATP synthase subunit 6 in human cells results in protein targeting to mitochondria but no assembly into ATP synthase

1 reference

12 December 2020

[Response to idebenone and multivitamin therapy in Leber's hereditary optic neuropathy]

1 reference

12 December 2020

Retinal ganglion cell neurodegeneration in mitochondrial inherited disorders

1 reference

12 December 2020

Limitations to in vivo import of hydrophobic proteins into yeast mitochondria. The case of a cytoplasmically synthesized apocytochrome b

1 reference

12 December 2020

Modulating mtDNA heteroplasmy by mitochondria-targeted restriction endonucleases in a 'differential multiple cleavage-site' model

1 reference

12 December 2020

Mitochondrial deafness

1 reference

12 December 2020

Mitochondrial dysfunction and biogenesis in the pathogenesis of Parkinson's disease.

1 reference

12 December 2020

Limitations of Allotopic Expression of Mitochondrial Genes in Mammalian Cells

1 reference

12 December 2020

Lentiviral transgenesis

1 reference

12 December 2020

Gene therapy for mitochondrial disease by delivering restriction endonuclease SmaI into mitochondria

1 reference

12 December 2020

Organ-specific shifts in mtDNA heteroplasmy following systemic delivery of a mitochondria-targeted restriction endonuclease

1 reference

12 December 2020

Opa1 deficiency in a mouse model of autosomal dominant optic atrophy impairs mitochondrial morphology, optic nerve structure and visual function

1 reference

12 December 2020

Suppression of mutations in mitochondrial DNA by tRNAs imported from the cytoplasm

1 reference

12 December 2020

tRNA mitochondrial import in yeast: Mapping of the import determinants in the carrier protein, the precursor of mitochondrial lysyl-tRNA synthetase

1 reference

12 December 2020

Case 15-2009: a man with coma after cardiac arrest

1 reference

12 December 2020

10.1007/S10545-010-9131-5

Identifiers

DOI

1 reference

22 August 2017

1 reference