Pharmacodynamic studies of a histone deacetylase inhibitor in Friedreich’s ataxia

EU Clinical Trials Register

EudraCT Number: 2011-002744-27
Sponsor's Protocol Code Number: CR01849
National Competent Authority: UK - MHRA
Clinical Trial Type: EEA CTA
Trial Status: Ongoing
Date on which this record was first entered in the EudraCT database: 2011-09-16

Ataxia UK, Lay summary of Festenstein's hdaci study

HSC20 interacts with Frataxin and is involved in iron-sulfur cluster biogenesis and iron homeostasis

Hum. Mol. Genet. (2011) doi: 10.1093/hmg/ddr582

Yuxi Shan and Gino Cortopassi
Molecular Biosciences, 1120 Haring Hall, University of California, Davis, CA 95616

Keywords: Friedreich’s ataxia (FRDA), frataxin, mitochondrial iron-sulfur cluster (ISC) biosynthesis, ISC assembly machinery, mitochondrial chaperone HSC20, iron-dependent, ISCU/Nfs1, GRP75 ISC chaperone.

Improved Histone Deacetylase Inhibitors as Therapeutics for the Neurodegenerative Disease Friedreich’s Ataxia: A New Synthetic Route

Pharmaceuticals 2011, 4, 1578-1590; doi:10.3390/ph4121578 (doi registration under processing)

Chunping Xu 1, Elisabetta Soragni 1, Vincent Jacques 2, James R. Rusche 2 and Joel M. Gottesfeld 1.
1 Department of Molecular Biology, The Scripps Research Institute, 10550 N. Torreys Pines Road, La Jolla, CA 92037, USA
2 Repligen Corporation, 41 Seyon Street, Waltham, MA 02453, USA

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Grant Award: Electrical activity in a stem cell model of Friedreich Ataxia.

The Brain Foundation.
Australian Brain Research - Dr Karina Needham from the University of Melbourne received the Friedreich Ataxia Grant Award for her project title: Electrical activity in a stem cell model of Friedreich Ataxia.

Epigenetic Enhancement of BDNF Signaling Rescues Synaptic Plasticity in Aging

The Journal of Neuroscience, 7 December 2011, 31(49): 17800-17810; doi: 10.1523/​JNEUROSCI.3878-11.2011

Yan Zeng, Miao Tan, Jun Kohyama, Marissa Sneddon, Joseph B. Watson, Yi E. Sun, and
Cui-Wei Xie
Department of Psychiatry and Biobehavioral Sciences, Semel Institute for Neuroscience and Human Behavior, University of California, Los Angeles, California 90024

Keywords: Aging-related cognitive declines, synaptic plasticity, dendritic spines, reduced histone acetylation, brain-derived neurotrophic factor (Bdnf) gene, H3 and H4 acetylation, HDAC inhibition, 7,8-dihydroxyflavone, trkB, epigenetic or pharmacological.

Substantia nigra hypoechogenicity in friedreich ataxia

Movement Disorders, Article first published online: 9 DEC 2011 | DOI: 10.1002/mds.23989

Heike Stockner MD, Martin Sojer MD, Sascha Hering MD, Wolfgang Nachbauer MD, Klaus Seppi MD, Christoph Schmidauer MD, Werner Poewe MD, Sylvia

Mutations in the dimer interface of dihydrolipoamide dehydrogenase promote site-specific oxidative damages in yeast and human cells

J. Biol. Chem. jbc.M111.274415. First Published on September 19, 2011, doi:10.1074/jbc.M111.274415

Rachael A. Vaubel, Pierre Rustin, and Grazia Isaya


DLD dimer interface mutations may accelerate frataxin turnover via an unknown mechanism

The role of DLD proteolytic activity remains unclear, and will require a better understanding of conditions that accelerate FXN turnover and the identification of additional natural substrates cleaved by DLD.

Structure-function study of cellular iron chemistry

OhioLINK ETD Center, Document number: osu1245414801

Huang, Jia

Keywords: iron-sulfur clusters, frataxin, ferrous iron, scaffold protein human ISU, cytosolic aconitase, IRP1, HscB, ITC.

The yeast metacaspase is implicated in oxidative stress response in frataxin-deficient cells

FEBS Letters - 07 December 2011 (10.1016/j.febslet.2011.12.002)

Sophie Lefevre, Dominika Sliwa, Françoise Auchère, Caroline Brossas, Christoph Ruckenstuhl, Nicole Boggetto, Emmanuel Lesuisse, Frank Madeo, Jean-Michel Camadro, Renata Santos

Keywords: Friedreich ataxia, mitochondrial frataxin, iron-sulfur cluster, heme biosynthesis, antioxidant therapies, hydrogen peroxide, metacaspase.

Posttranslational Modification of Cysteine in Redox Signaling and Oxidative Stress – Focus on S-Glutathionylation

Antioxidants & Redox Signaling. null, Vol. 0, No. ja

Prof. John J. Mieyal, Case Western Reserve University, School of Medicine, Pharmacology, Cleveland, Ohio, United States.
Boon Chock, NHLBI, National Institutes of Health, Laboratory of Biochemistry, Bethesda, Maryland, United States.

Keywords: Reactive oxygen species (ROS), reactive nitrogen species (RNS), cellular signaling pathways, peroxynitrite, cysteine residues, reversible protein-S-glutathionylation, mitochondria, cardiovascular system, neurodegenerative diseases.