Prolonged treatment with pimelic o-aminobenzamide HDAC inhibitors ameliorates the disease phenotype of a Friedreich ataxia mouse model

Neurobiology of Disease, doi:10.1016/j.nbd.2011.02.016

Chiranjeevi Sandi a, Ricardo Mouro Pinto a, 1, Sahar Al-Mahdawi a, Vahid Ezzatizadeh a, Glenn Barnes b, Steve Jones b, James R. Rusche b, Joel M. Gottesfeld c and Mark A. Pook a

a Division of Biosciences, School of Health Sciences and Social Care, Brunel University, Uxbridge, UB8 3PH, UK
b Repligen Corporation, Waltham, MA, USA
c Department of Molecular Biology, The Scripps Research Institute, La Jolla, CA, USA
Available online 10 March 2011.

keywords: Friedreich ataxia (FRDA), Histone deacetylase (HDAC) inhibitors, pimelic o-aminobenzamide compounds 106, 109 and 136, FXN gene silencing, short-term studies, long-term therapeutic effects, FRDA mouse model.

Clinical proof of concept patient trials for OX1 in Friedreich's Ataxia

Intellect Neurosciences, Inc. is a Manhattan-based biopharmaceutical company
The Company's most advanced internally developed product is OX1 which has been tested in Phase 1 clinical trials. The Company plans to conduct clinical proof of concept patient trials for OX1 in Alzheimer's disease and Friedreich's Ataxia,

New Robot To Help People To Walk Again

Medical News Today, Article Date: 09 Mar 2011

"Over the four-year period, the project will produce two demonstrators, among them a novel mobile robot-assisted gait rehabilitation system which will be a self-aware system capable of learning to enable it to optimally match the requirements of the user at different stages of rehabilitation."

Regulation of chromatin by histone modifications

Cell Research (2011) 21:381–395. doi:10.1038/cr.2011.22;

Andrew J Bannister1 and Tony Kouzarides1

1The Gurdon Institute and Department of Pathology, University of Cambridge, Cambridge CB2 1QN, UK

Keywords:

histone; modifications; chromatin

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Chemoproteomics profiling of HDAC inhibitors reveals selective targeting of HDAC complexes

Nature Biotechnology, Year published:(2011), doi:10.1038/nbt.1759

1. Cellzome AG, Heidelberg, Germany.
Marcus Bantscheff, Carsten Hopf, Mikhail M Savitski, Antje Dittmann, Paola Grandi, Anne-Marie Michon, Judith Schlegl, Yann Abraham, Isabelle Becher, Giovanna Bergamini, Markus Boesche, Manja Delling, Birgit Dümpelfeld, Dirk Eberhard, Carola Huthmacher, Toby Mathieson, Daniel Poeckel, Katja Strunk, Gavain Sweetman, Ulrich Kruse, Gitte Neubauer & Gerard Drewes
2. Cellzome Ltd., Chesterford Research Park, Cambridge, United Kingdom.
Valérie Reader & Nigel G Ramsden

Frataxin participates to the hypoxia-induced response in tumors

Cell Death and Disease (2011) 2, e123; doi:10.1038/cddis.2011.5

Guccini I, Serio D, Condò I, Rufini A, Tomassini B, Mangiola A, Maira G, Anile C, Fina D, Pallone F, Mongiardi MP, Levi A, Ventura N, Testi R, Malisan F

Keywords: frataxin, Friedreich's ataxia, oxidative stress, apoptosis, tumor suppressor, stress resistance, tumor cells, response to hypoxic stress, hypoxia-inducible factors (HIF), tumor-suppressor p53.

"frataxin is in fact increased in human tumors in vivo"
"Frataxin upregulation in response to hypoxia is dependent on hypoxia-inducible factors expression"

Cardiomyopathy of Friedreich's Ataxia: Use of Mouse Models to Understand Human Disease and Guide Therapeutic Development.

Pediatr Cardiol. 2011 Mar 1. [Epub ahead of print]

Payne RM, Pride PM, Babbey CM.

Riley Heart Research Center, Wells Center for Pediatric Research, Indiana University School of Medicine, 1044 West Walnut, R4302, Indianapolis, IN, 46202, USA

Keywords: Friedreich's ataxia, heart, brain, severe cardiomyopathy, heart failure, frataxin, Animal models, to develop new therapeutic strategies.

Brain diffusion-weighted imaging in Friedreich's ataxia

Mov Disord. 2011 Mar 2. doi: 10.1002/mds.23518.

Rizzo G, Tonon C, Valentino ML, Manners D, Fortuna F, Gellera C, Pini A, Ghezzo A, Baruzzi A, Testa C, Malucelli E, Barbiroli B, Carelli V, Lodi R.

MR Spectroscopy Unit, Department of Internal Medicine, Aging and Nephrology, University of Bologna, Bologna, Italy; Department of Neurological Sciences, University of Bologna, Bologna, Italy.

"The extent of neurodegeneration in FRDA appears more extended than previously reported."

Keywords: Friedreich ataxia (FRDA), brain damage in FRDA, in vivo markers of neurodegeneration, diffusion-weighted imaging (DWI), Axial DW images, mean diffusivity (MD) maps, medulla, pons, inferior, middle and superior cerebellar peduncles (ICP, SCP, MCP), dentate nucleus, cerebellar white matter, thalamus, caudate, putamen, pallidus, pyramidal tracts at level of posterior limb of internal capsule (PLIC), optic radiations (OR), and corpus callosum, Histograms of MD, International Cooperative Ataxia Rating Scale (ICARS).

Impaired inhibition of prepotent motor tendencies in Friedreich ataxia demonstrated by the Simon interference task.

Brain Cogn. 2011 Feb 25

Corben LA, Akhlaghi H, Georgiou-Karistianis N, Bradshaw JL, Egan GF, Storey E, Churchyard AJ, Delatycki MB.

Bruce Lefroy Centre for Genetic Health Research, Murdoch Childrens Research Institute, Parkville, Victoria, Australia; Experimental Neuropsychology Research Unit, School of Psychology and Psychiatry, Monash University, Clayton 3800, Victoria, Australia.

KEYWORDS: Friedreich ataxia (FRDA), impairment in motor planning, cerebral cortex and/or cerebello-cortical projections, Simon interference task.

Human Basal Ganglia and the Dynamic Control of Force during On-Line Corrections

The Journal of Neuroscience, February 2, 2011, 31(5):1600-1605; doi:10.1523/JNEUROSCI.3301-10.2011

Scott T. Grafton1 and Eugene Tunik2

1Sage Center for the Study of Mind, Department of Psychology, University of California, Santa Barbara, California 93106, and 2Department of Rehabilitation and Movement Science, University of Medicine and Dentistry of New Jersey, Newark, New Jersey 07107

Keywords: Natural movements, submovements, basal ganglia, force amplitude, change, and duration, proprioceptive error, globus pallidus, subthalamic nucleus.