Advanced search
Publication Cover
Expert Opinion on Investigational Drugs Volume 12, 2003 - Issue 2
Submit an article Journal homepage
139
Views
62
CrossRef citations to date
0
Altmetric
Review

Friedreich’s ataxia: iron chelators that target the mitochondrion as a therapeutic strategy?

DR Richardson Iron Metabolism and Chelation Program, Children’s Cancer Institute Australia for Medical Research, High St (PO Box 81), Randwick, Sydney, New South Wales, 2031, Australia. [email protected]
Pages 235-245 | Published online: 02 Mar 2005

Citations (62)

Keep up to date with the latest research on this topic with citation updates for this article.

Subscribe to citation updates

Read on this site (5)

Michelangelo Mancuso, Daniele Orsucci, Anna Choub & Gabriele Siciliano. (2010) Current and emerging treatment options in the management of Friedreich ataxia. Neuropsychiatric Disease and Treatment 6, pages 491-499.
Read now
Eugene D. Weinberg. (2008) Iron Out-of-Balance: A Risk Factor for Acute and Chronic Diseases. Hemoglobin 32:1-2, pages 117-122.
Read now
E. D. Weinberg. (2007) Iron loading in humans: A risk factor for enhanced morbidity and mortality. Journal of Nutritional & Environmental Medicine 16:1, pages 43-51.
Read now
David B. Lovejoy, Danuta Kalinowski, Paul V. Bernhardt & Des R. Richardson. (2006) PCTH: A Novel Orally Active Chelator for the Treatment of Iron Overload Disease. Hemoglobin 30:1, pages 93-104.
Read now
Susan L Perlman. (2003) Spinocerebellar degeneration. Expert Opinion on Pharmacotherapy 4:10, pages 1637-1641.
Read now

Articles from other publishers (57)

William Motley, Vinay Chaudry & Thomas E. Lloyd. 2022. Neuromuscular Disorders. Neuromuscular Disorders 278 311 .
Daphne S. Cukierman, Beatriz N. Evangelista, Carlos Castanho Neto, Chris H.J. Franco, Luiz Antônio S. Costa, Renata Diniz, Jones Limberger & Nicolás A. Rey. (2021) Mildness in preparative conditions directly affects the otherwise straightforward syntheses outcome of Schiff-base isoniazid derivatives: Aroylhydrazones and their solvolysis-related dihydrazones. Journal of Molecular Structure 1228, pages 129437.
Crossref
Shannon Chiang, Danuta S. Kalinowski, Mahendiran Dharmasivam, Nady Braidy, Des R. Richardson & Michael L.H. Huang. (2020) The potential of the novel NAD+ supplementing agent, SNH6, as a therapeutic strategy for the treatment of Friedreich’s ataxia. Pharmacological Research 155, pages 104680.
Crossref
Elizabeth W. Hunsaker & Katherine J. Franz. (2019) Emerging Opportunities To Manipulate Metal Trafficking for Therapeutic Benefit. Inorganic Chemistry 58:20, pages 13528-13545.
Crossref
Diane M. Ward & Suzanne M. Cloonan. (2019) Mitochondrial Iron in Human Health and Disease. Annual Review of Physiology 81:1, pages 453-482.
Crossref
Elisia ClarkJoseph JohnsonYi Na DongElizabeth Mercado-AyonNathan WarrenMattieu ZhaiEmily McMillanAmy SalovinHong LinDavid R. Lynch. (2018) Role of frataxin protein deficiency and metabolic dysfunction in Friedreich ataxia, an autosomal recessive mitochondrial disease. Neuronal Signaling 2:4.
Crossref
Marco Nuñez & Pedro Chana-Cuevas. (2018) New Perspectives in Iron Chelation Therapy for the Treatment of Neurodegenerative Diseases. Pharmaceuticals 11:4, pages 109.
Crossref
Shannon Chiang, Zaklina Kovacevic, Sumit Sahni, Darius J.R. Lane, Angelica M. Merlot, Danuta S. Kalinowski, Michael L.-H. Huang & Des R. Richardson. (2016) Frataxin and the molecular mechanism of mitochondrial iron-loading in Friedreich's ataxia. Clinical Science 130:11, pages 853-870.
Crossref
Yee-Ki Lee, Yee-Man Lau, Kwong-Man Ng, Wing-Hon Lai, Shu-Leong Ho, Hung-Fat Tse, Chung-Wah Siu & Philip Wing-Lok Ho. (2016) Efficient attenuation of Friedreich's ataxia (FRDA) cardiomyopathy by modulation of iron homeostasis-human induced pluripotent stem cell (hiPSC) as a drug screening platform for FRDA. International Journal of Cardiology 203, pages 964-971.
Crossref
F. Weidemann, F. Scholz, C. Florescu, D. Liu, K. Hu, S. Herrmann, G. Ertl & S. Störk. (2014) Herzbeteiligung bei Friedreich-AtaxieHeart involvement in Friedreich’s ataxia. Herz 40:S1, pages 85-90.
Crossref
H. Puccio, M. Anheim & C. Tranchant. (2014) Pathophysiogical and therapeutic progress in Friedreich ataxia. Revue Neurologique 170:5, pages 355-365.
Crossref
A Anzovino, D J R Lane, M L-H Huang & D R Richardson. (2014) Fixing frataxin: ‘ironing out’ the metabolic defect in Friedreich's ataxia. British Journal of Pharmacology 171:8, pages 2174-2190.
Crossref
Frank Weidemann, Stefan Störk, Dan Liu, Kai Hu, Sebastian Herrmann, Georg Ertl & Markus Niemann. (2013) Cardiomyopathy of Friedreich Ataxia. Journal of Neurochemistry 126, pages 88-93.
Crossref
Annalisa Pastore. 2013. Mechanisms and Metal Involvement in Neurodegenerative Diseases. Mechanisms and Metal Involvement in Neurodegenerative Diseases 98 117 .
Timothy E. Richardson, Heather N. Kelly, Amanda E. Yu & James W. Simpkins. (2013) Therapeutic strategies in Friedreich's Ataxia. Brain Research 1514, pages 91-97.
Crossref
Amy K. Rines & Hossein Ardehali. (2013) Transition metals and mitochondrial metabolism in the heart. Journal of Molecular and Cellular Cardiology 55, pages 50-57.
Crossref
Marina Bayeva, Mihai Gheorghiade & Hossein Ardehali. (2013) Mitochondria as a Therapeutic Target in Heart Failure. Journal of the American College of Cardiology 61:6, pages 599-610.
Crossref
Robert B. Wilson. (2012) Therapeutic Developments in Friedreich Ataxia. Journal of Child Neurology 27:9, pages 1212-1216.
Crossref
Robert Sutak, Alexandra Seguin, Ricardo Garcia‐Serres, Jean‐Louis Oddou, Andrew Dancis, Jan Tachezy, Jean‐Marc Latour, Jean‐Michel Camadro & Emmanuel Lesuisse. (2012) Human mitochondrial ferritin improves respiratory function in yeast mutants deficient in iron–sulfur cluster biogenesis, but is not a functional homologue of yeast frataxin. MicrobiologyOpen 1:2, pages 95-104.
Crossref
Piyush M. Vyas, Wendy J. Tomamichel, P. Melanie Pride, Clifford M. Babbey, Qiujuan Wang, Jennifer Mercier, Elizabeth M. Martin & R. Mark Payne. (2012) A TAT–Frataxin fusion protein increases lifespan and cardiac function in a conditional Friedreich's ataxia mouse model. Human Molecular Genetics 21:6, pages 1230-1247.
Crossref
Alain Martelli, Marek Napierala & Hélène Puccio. (2012) Understanding the genetic and molecular pathogenesis of Friedreich’s ataxia through animal and cellular models. Disease Models & Mechanisms 5:2, pages 165-176.
Crossref
Christer B. Aakeröy, Safiyyah Forbes & John Desper. (2012) The effect of water molecules in stabilizing co-crystals of active pharmaceutical ingredients. CrystEngComm 14:7, pages 2435.
Crossref
Susan L. Perlman. 2012. Ataxic Disorders. Ataxic Disorders 635 654 .
Daniele Marmolino. (2011) Friedreich's ataxia: Past, present and future. Brain Research Reviews 67:1-2, pages 311-330.
Crossref
R. Mark Payne. (2011) The heart in Friedreich's Ataxia: Basic findings and clinical implications. Progress in Pediatric Cardiology 31:2, pages 103-109.
Crossref
Daniel Velasco-Sánchez, Asuncion Aracil, Raquel Montero, Ana Mas, Lorenzo Jiménez, Mar O’Callaghan, Maria Tondo, Antoni Capdevila, Josep Blanch, Rafael Artuch & Mercedes Pineda. (2010) Combined Therapy with Idebenone and Deferiprone in Patients with Friedreich’s Ataxia. The Cerebellum 10:1, pages 1-8.
Crossref
R. Mark Payne, P. Melanie Pride & Clifford M. Babbey. (2011) Cardiomyopathy of Friedreich’s Ataxia: Use of Mouse Models to Understand Human Disease and Guide Therapeutic Development. Pediatric Cardiology 32:3, pages 366-378.
Crossref
Renata SantosSophie LefevreDominika SliwaAlexandra SeguinJean-Michel CamadroEmmanuel Lesuisse. (2010) Friedreich Ataxia: Molecular Mechanisms, Redox Considerations, and Therapeutic Opportunities. Antioxidants & Redox Signaling 13:5, pages 651-690.
Crossref
M. G. Efimova & Y. Trottier. (2010) Distribution of frataxin in eye retina of normal mice and of transgenic R7E mice with retinal degeneration. Journal of Evolutionary Biochemistry and Physiology 46:4, pages 414-417.
Crossref
Ana R. Correia, Tao WangElizabeth A. CraigCláudio M. Gomes. (2010) Iron-binding activity in yeast frataxin entails a trade off with stability in the α1/β1 acidic ridge region. Biochemical Journal 426:2, pages 197-203.
Crossref
Eugene D. Weinberg. (2010) The hazards of iron loading. Metallomics 2:11, pages 732.
Crossref
Nadège Calmels, Hervé Seznec, Pascal Villa, Laurence Reutenauer, Marcel Hibert, Jacques Haiech, Pierre Rustin, Michel Koenig & Hélène Puccio. (2009) Limitations in a frataxin knockdown cell model for Friedreich ataxia in a high-throughput drug screen. BMC Neurology 9:1.
Crossref
Daniele Marmolino & Fabio Acquaviva. (2009) Friedreich’s Ataxia: From the (GAA) n Repeat Mediated Silencing to New Promising Molecules for Therapy. The Cerebellum 8:3, pages 245-259.
Crossref
Michael D. Hebert. (2008) Targeting the gene in Friedreich ataxia. Biochimie 90:8, pages 1131-1139.
Crossref
Megan WhitnallYohan Suryo RahmantoRobert SutakXiangcong XuErika M. BeckerMarc R. Mikhael, Prem Ponka & Des R. Richardson. (2008) The MCK mouse heart model of Friedreich's ataxia: Alterations in iron-regulated proteins and cardiac hypertrophy are limited by iron chelation. Proceedings of the National Academy of Sciences 105:28, pages 9757-9762.
Crossref
Tanja Schmitz-Hübsch & Thomas Klockgether. (2008) An update on inherited ataxias. Current Neurology and Neuroscience Reports 8:4, pages 310-319.
Crossref
K. Li, E. K. Besse, D. Ha, G. Kovtunovych & T. A. Rouault. (2008) Iron-dependent regulation of frataxin expression: implications for treatment of Friedreich ataxia. Human Molecular Genetics 17:15, pages 2265-2273.
Crossref
Robert Sutak, Xiangcong Xu, Megan Whitnall, Mohammed Abul Kashem, Daniel Vyoral & Des R. Richardson. (2008) Proteomic analysis of hearts from frataxin knockout mice: Marked rearrangement of energy metabolism, a response to cellular stress and altered expression of proteins involved in cell structure, motility and metabolism. PROTEOMICS 8:8, pages 1731-1741.
Crossref
Joel M. Gottesfeld. (2007) Small molecules affecting transcription in Friedreich ataxia. Pharmacology & Therapeutics 116:2, pages 236-248.
Crossref
Xi-Ping Huang, Peter J. O’Brien & Douglas M. Templeton. (2006) Mitochondrial involvement in genetically determined transition metal toxicity. Chemico-Biological Interactions 163:1-2, pages 68-76.
Crossref
Megan Whitnall & Des R. Richardson. (2006) Iron: A New Target for Pharmacological Intervention in Neurodegenerative Diseases. Seminars in Pediatric Neurology 13:3, pages 186-197.
Crossref
Robert B. Wilson. (2006) Iron Dysregulation in Friedreich Ataxia. Seminars in Pediatric Neurology 13:3, pages 166-175.
Crossref
Raffaele LodiCaterina TononVittorio CalabreseAnthony H.V. Schapira. (2006) Friedreich's Ataxia: From Disease Mechanisms to Therapeutic Interventions. Antioxidants & Redox Signaling 8:3-4, pages 438-443.
Crossref
Bennett Van Houten, Victoria Woshner & Janine H. Santos. (2006) Role of mitochondrial DNA in toxic responses to oxidative stress. DNA Repair 5:2, pages 145-152.
Crossref
Danuta S. Kalinowski & Des R. Richardson. (2005) The Evolution of Iron Chelators for the Treatment of Iron Overload Disease and Cancer. Pharmacological Reviews 57:4, pages 547-583.
Crossref
Hava GlicksteinRinat Ben ElMaya ShvartsmanZ. Ioav Cabantchik. (2005) Intracellular labile iron pools as direct targets of iron chelators: a fluorescence study of chelator action in living cells. Blood 106:9, pages 3242-3250.
Crossref
Paul V. Bernhardt, Piao Chin, Philip C. Sharpe, Jing-Yan C. Wang & Des R. Richardson. (2005) Novel diaroylhydrazine ligands as iron chelators: coordination chemistry and biological activity. JBIC Journal of Biological Inorganic Chemistry 10:7, pages 761-777.
Crossref
Vittorio Calabrese, Raffaele Lodi, Caterina Tonon, Velia D'Agata, Maria Sapienza, Giovanni Scapagnini, Andrea Mangiameli, Giovanni Pennisi, A.M. Giuffrida Stella & D. Allan Butterfield. (2005) Oxidative stress, mitochondrial dysfunction and cellular stress response in Friedreich's ataxia. Journal of the Neurological Sciences 233:1-2, pages 145-162.
Crossref
Martin B DelatyckiPanos A IoannouAndrew J Churchyard. (2005) Friedreich ataxia: from genes to therapies?. Medical Journal of Australia 182:9, pages 439-439.
Crossref
Eylon Yavin, Raghavendra Kikkiri, Shosh Gil, Rina Arad-Yellin, Ephraim Yavin & Abraham Shanzer. (2005) Synthesis and biological evaluation of lipophilic iron chelators as protective agents from oxidative stress. Organic & Biomolecular Chemistry 3:15, pages 2685.
Crossref
R.H.B. Meyboom. 2005. Side Effects of Drugs Annual 28. Side Effects of Drugs Annual 28 254 260 .
Susan L. Perlman. (2004) Symptomatic and Disease-Modifying Therapy for the Progressive Ataxias. The Neurologist 10:5, pages 275-289.
Crossref
Madhavi Thomas & Joseph Jankovic. (2004) Neurodegenerative disease and iron storage in the brain. Current Opinion in Neurology 17:4, pages 437-442.
Crossref
DES R. RICHARDSON. (2004) Novel Chelators for Central Nervous System Disorders That Involve Alterations in the Metabolism of Iron and Other Metal Ions. Annals of the New York Academy of Sciences 1012:1, pages 326-341.
Crossref
Hervé Seznec, Robert B Wilson & Hélène Puccio. (2004) 2003 International Friedreich's Ataxia Research Conference, 14–16 February 2003, Bethesda, MD, USA. Neuromuscular Disorders 14:1, pages 70-82.
Crossref
L. Schöls, Ch. Meyer, G. Schmid, I. Wilhelms & H. Przuntek. 2004. Focus on Extrapyramidal Dysfunction. Focus on Extrapyramidal Dysfunction 135 145 .
Gang Liu, Ping Men, George Perry & Mark A. Smith. 2007. Nanotechnologies for the Life Sciences. Nanotechnologies for the Life Sciences.

Reprints and Corporate Permissions

Please note: Selecting permissions does not provide access to the full text of the article, please see our help page How do I view content?

To request a reprint or corporate permissions for this article, please click on the relevant link below:

Order Reprints Request Corporate Permissions

Academic Permissions

Please note: Selecting permissions does not provide access to the full text of the article, please see our help page How do I view content?

Obtain permissions instantly via Rightslink by clicking on the button below:

Request Academic Permissions

If you are unable to obtain permissions via Rightslink, please complete and submit this Permissions form. For more information, please visit our Permissions help page.

Related research

People also read lists articles that other readers of this article have read.

Recommended articles lists articles that we recommend and is powered by our AI driven recommendation engine.

Cited by lists all citing articles based on Crossref citations.
Articles with the Crossref icon will open in a new tab.