Drug development for mitochondrial disease: recent progress, current challenges, and future prospects

Genetic and biochemical intricacy shapes mitochondrial cytopathies

(2016) Douglass M. Turnbull et al.   NEUROBIOLOGY OF DISEASE

Carvacrol inhibits proliferation and induces apoptosis in human colon cancer cells

(2015) Kai Fan et al.   ANTI-CANCER DRUGS

Mitochondrial Sirtuins and Their Relationships with Metabolic Disease and Cancer

(2015) Surinder Kumar et al.   ANTIOXIDANTS & REDOX SIGNALING

An Essential Role of the Mitochondrial Electron Transport Chain in Cell Proliferation Is to Enable Aspartate Synthesis

(2015) Kıvanç Birsoy et al.   CELL

Supporting Aspartate Biosynthesis Is an Essential Function of Respiration in Proliferating Cells

(2015) Lucas B. Sullivan et al.   CELL

The link between mitochondrial complex I and brain-derived neurotrophic factor in SH-SY5Y cells – The potential of JNX1001 as a therapeutic agent

(2015) Helena K. Kim et al.   EUROPEAN JOURNAL OF PHARMACOLOGY

Natural Compounds Modulating Mitochondrial Functions

(2015) Lara Gibellini et al.   Evidence-based Complementary and Alternative Medicine

Polydatin protects hepatocytes against mitochondrial injury in acute severe hemorrhagic shock via SIRT1-SOD2pathway

(2015) Pengyun Li et al.   EXPERT OPINION ON THERAPEUTIC TARGETS

Emerging aspects of treatment in mitochondrial disorders

(2015) Shamima Rahman   JOURNAL OF INHERITED METABOLIC DISEASE

Mitochondrial Carnitine/Acylcarnitine Translocase: Insights in Structure/ Function Relationships. Basis for Drug Therapy and Side Effects Prediction

(2015) Annamaria Tonazzi et al.   MINI-REVIEWS IN MEDICINAL CHEMISTRY

Keep the fire burning: Current avenues in the quest of treating mitochondrial disorders

(2015) Christin Tischner et al.   MITOCHONDRION

Administration of CoQ10 analogue ameliorates dysfunction of the mitochondrial respiratory chain in a mouse model of Angelman syndrome

(2015) Katrina J. Llewellyn et al.   NEUROBIOLOGY OF DISEASE

The Roles of PINK1, Parkin, and Mitochondrial Fidelity in Parkinson’s Disease

(2015) Alicia M. Pickrell et al.   NEURON

Mitochondrial Dysfunction Contributes to the Pathogenesis of Alzheimer’s Disease

(2015) Fabian A. Cabezas-Opazo et al.   Oxidative Medicine and Cellular Longevity

Protection against renal ischemia–reperfusion injury in vivo by the mitochondria targeted antioxidant MitoQ

(2015) Anna J. Dare et al.   Redox Biology

Nuclear expression of mitochondrial ND4 leads to the protein assembling in complex I and prevents optic atrophy and visual loss

(2015) Hélène Cwerman-Thibault et al.   Molecular Therapy-Methods & Clinical Development

NAD+-Dependent Activation of Sirt1 Corrects the Phenotype in a Mouse Model of Mitochondrial Disease

(2014) Raffaele Cerutti et al.   Cell Metabolism

Gene therapy for mitochondrial diseases: Leber Hereditary Optic Neuropathy as the first candidate for a clinical trial

(2014) Hélène Cwerman-Thibault et al.   COMPTES RENDUS BIOLOGIES

Physiological Effect and Therapeutic Application of Alpha Lipoic Acid

(2014) Sungmi Park et al.   CURRENT MEDICINAL CHEMISTRY

Monitoring Cardiac Function During Idebenone Therapy in Friedreich's Ataxia

(2014) Di Giovanni et al.   CURRENT PHARMACEUTICAL DESIGN

Editorial (Thematic Issue: Mitochondrial Biogenesis: Pharmacological Approaches)

(2014) Teresa Valero   CURRENT PHARMACEUTICAL DESIGN

Pioglitazone treatment restoresin vivomuscle oxidative capacity in a rat model of diabetes

(2014) B. Wessels et al.   DIABETES OBESITY & METABOLISM

Salvaging hope: Is increasing NAD+ a key to treating mitochondrial myopathy?

(2014) R. N. Lightowlers et al.   EMBO Molecular Medicine

LHON Gene Therapy Vector Prevents Visual Loss and Optic Neuropathy Induced by G11778A Mutant Mitochondrial DNA: Biodistribution and Toxicology Profile

(2014) R. Koilkonda et al.   INVESTIGATIVE OPHTHALMOLOGY & VISUAL SCIENCE

Treatment of Mitochondrial Disorders

(2014) Gregory M. Enns   JOURNAL OF CHILD NEUROLOGY

Ketogenic diets, mitochondria, and neurological diseases

(2014) Lindsey B. Gano et al.   JOURNAL OF LIPID RESEARCH

Riboflavin and migraine: the bridge over troubled mitochondria

(2014) Bruno Colombo et al.   NEUROLOGICAL SCIENCES

The Target of Metformin in Type 2 Diabetes

(2014) Ele Ferrannini   NEW ENGLAND JOURNAL OF MEDICINE

Thiamine transporter-2 deficiency: outcome and treatment monitoring

(2014) Juan Ortigoza-Escobar et al.   Orphanet Journal of Rare Diseases

Rosiglitazone Causes Cardiotoxicity via Peroxisome Proliferator-Activated Receptor γ-Independent Mitochondrial Oxidative Stress in Mouse Hearts

(2014) Huamei He et al.   TOXICOLOGICAL SCIENCES

Increasing Superoxide Production and the Labile Iron Pool in Tumor Cells may Sensitize Them to Extracellular Ascorbate

(2014) Mark Frederick McCarty et al.   Frontiers in Oncology

Modelling biochemical features of mitochondrial neuropathology

(2013) Matthew J. Bird et al.   BIOCHIMICA ET BIOPHYSICA ACTA-GENERAL SUBJECTS

Persistence of the treatment effect of idebenone in Leber’s hereditary optic neuropathy

(2013) T. Klopstock et al.   BRAIN

Turn up the power - pharmacological activation of mitochondrial biogenesis in mouse models

(2013) J C Komen et al.   BRITISH JOURNAL OF PHARMACOLOGY

Pioglitazone alleviates the mitochondrial apoptotic pathway and mito-oxidative damage in thed-galactose-induced mouse model

(2013) Atish Prakash et al.   CLINICAL AND EXPERIMENTAL PHARMACOLOGY AND PHYSIOLOGY

Co-enzyme Q10and idebenone use in Friedreich's ataxia

(2013) Michael H Parkinson et al.   JOURNAL OF NEUROCHEMISTRY

Review of Clinical Trials for Mitochondrial Disorders: 1997–2012

(2013) Douglas S. Kerr   Neurotherapeutics

Tissue- and cell-type-specific manifestations of heteroplasmic mtDNA 3243A>G mutation in human induced pluripotent stem cell-derived disease model

(2013) R. H. Hamalainen et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

mTOR Inhibition Alleviates Mitochondrial Disease in a Mouse Model of Leigh Syndrome

(2013) S. C. Johnson et al.   SCIENCE

Zebrafish as a model system for mitochondrial biology and diseases

(2013) Shelby L. Steele et al.   Translational Research

Is Leber Hereditary Optic Neuropathy Treatable? Encouraging Results With Idebenone in Both Prospective and Retrospective Trials and An Illustrative Case

(2012) Esfandiar J. Sabet-Peyman et al.   JOURNAL OF NEURO-OPHTHALMOLOGY

Impaired Short-Term Plasticity in Mossy Fiber Synapses Caused by Mitochondrial Dysfunction of Dentate Granule Cells Is the Earliest Synaptic Deficit in a Mouse Model of Alzheimer's Disease

(2012) S. H. Lee et al.   JOURNAL OF NEUROSCIENCE

RETRACTED: Bezafibrate improves mitochondrial function in the CNS of a mouse model of mitochondrial encephalopathy

(2012) Natalie Noe et al.   MITOCHONDRION

EPI-743 reverses the progression of the pediatric mitochondrial disease—Genetically defined Leigh Syndrome

(2012) Diego Martinelli et al.   MOLECULAR GENETICS AND METABOLISM

Animal models: Not close enough

(2012) Jocelyn Rice   NATURE

Idebenone in Friedreich ataxia cardiomyopathy—results from a 6-month phase III study (IONIA)

(2011) Sarah J. Lagedrost et al.   AMERICAN HEART JOURNAL

In Vivo Correction of COX Deficiency by Activation of the AMPK/PGC-1α Axis

(2011) Carlo Viscomi et al.   Cell Metabolism

Assessment of neurological efficacy of idebenone in pediatric patients with Friedreich's ataxia: data from a 6-month controlled study followed by a 12-month open-label extension study

(2011) Thomas Meier et al.   JOURNAL OF NEUROLOGY

Pharmacological targeting of mitochondrial complex I deficiency: The cellular level and beyond

(2011) Peggy Roestenberg et al.   MITOCHONDRION

A Phase 3, Double-blind, Placebo-Controlled Trial of Idebenone in Friedreich Ataxia

(2010) David R. Lynch et al.   ARCHIVES OF NEUROLOGY

Genetic background influences mitochondrial function: modeling mitochondrial disease for therapeutic development

(2010) Paule Bénit et al.   TRENDS IN MOLECULAR MEDICINE

Expression of the Ciona intestinalis Alternative Oxidase (AOX) in Drosophila Complements Defects in Mitochondrial Oxidative Phosphorylation

(2009) Daniel J.M. Fernandez-Ayala et al.   Cell Metabolism

Use of Tris-hydroxymethyl aminomethane in severe lactic acidosis due to highly active antiretroviral therapy: a case report

(2008) K. Marfo et al.   JOURNAL OF CLINICAL PHARMACY AND THERAPEUTICS

The Variability of the Harlequin Mouse Phenotype Resembles that of Human Mitochondrial-Complex I-Deficiency Syndromes

(2008) Paule Bénit et al.   PLoS One