Therapeutic Strategies in Friedreich's Ataxia

Therapeutic Strategies in Friedreich's Ataxia. Timothy E. Richardson, Heather N. Kelly, Amanda E. Yu, James W. Simpkins; Brain Research, Available online 13 April 2013. http://dx.doi.org/10.1016/j.brainres.2013.04.005

Keywords: Friedreich's ataxia (FA), FXN, frataxin, therapeutic research, ongoing treatment strategies, 17β-estradiol, methylene blue.

Cardiomyopathy and the electrocardiogram in Friedreich's ataxia

Cardiomyopathy and the electrocardiogram in Friedreich's ataxia . Alexandra H Wood, Simon W Dubrey; British Journal of Hospital Medicine, Vol. 74, Iss. 4, 09 Apr 2013, pp 232 - 233

Iron uptake in quiescent and inflammation-activated astrocytes: A potentially neuroprotective control of iron burden

Iron uptake in quiescent and inflammation-activated astrocytes: A potentially neuroprotective control of iron burden. Ilaria Pelizzoni, Daniele Zacchetti, Alessandro Campanella, Fabio Grohovaz, Franca Codazzi; Biochimica et Biophysica Acta (BBA) - Molecular Basis of Disease, Available online 11 April 2013. http://dx.doi.org/10.1016/j.bbadis.2013.04.007

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Keywords: Astrocytes, NTBI, iron uptake, DMT1, activation process, TRP channels, neuroinflammation

Vitamin Switches on a Gene, A Potential New Treatment for Friedreich's Ataxia?

Vitamin Switches on a Gene, A Potential New Treatment for Friedreich's Ataxia?. MRC Clinical Sciences Centre, Faculty of Medicine, Imperial College London.

Novel research from the CSC holds promise for a new therapeutic approach, using a commonly available vitamin supplement to modify the epigenetic controls of the genetic defect causing the illness.

Related to: Heterochromatinization induced by GAA-repeat hyperexpansion in Friedreich's ataxia can be reduced upon HDAC inhibition by Vitamin B3

Frataxin Deficiency Leads to Reduced Expression and Impaired Translocation of NF-E2-Related Factor (Nrf2) in Cultured Motor Neurons

Frataxin Deficiency Leads to Reduced Expression and Impaired Translocation of NF-E2-Related Factor (Nrf2) in Cultured Motor Neurons. D'Oria, V.; Petrini, S.; Travaglini, L.; Priori, C.; Piermarini, E.; Petrillo, S.; Carletti, B.; Bertini, E.; Piemonte, F.; International Journal of Molecular Sciences. 2013; 14(4):7853-7865.

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Mitochondria targeted therapeutic approaches in Parkinson's and Huntington's diseases

Mitochondria targeted therapeutic approaches in Parkinson's and Huntington's diseases. Rajnish K. Chaturvedi, M. Flint Beal. Molecular and Cellular Neuroscience, Volume 55, July 2013, Pages 101-114. http://dx.doi.org/10.1016/j.mcn.2012.11.011

Keywords: Parkinson's disease, Huntington's disease, Neurodegenerative diseases, Mitochondrial dysfunction, Creatine, Co-Q10, PGC-1α, Sirtuins.

Cerebello-cerebral connectivity deficits in Friedreich ataxia

Cerebello-cerebral connectivity deficits in Friedreich ataxia. Andrew Zalesky, Hamed Akhlaghi, Louise A. Corben, John L. Bradshaw, Martin B. Delatycki, Elsdon Storey, Nellie Georgiou-Karistianis, Gary F. Egan. Brain Structure and Function, April 2013. DOI 10.1007/s00429-013-0547-1

Keywords: Friedreich ataxia, Diffusion-weighted imaging, White matter, Connectome, Connectivity, supplementary motor area, cingulate cortex, frontal cortices, putamen, other subcortical nuclei.

Mitochondrial Diseases of the Brain

Mitochondrial Diseases of the Brain. Rajnish K. Chaturvedi, M. Flint Beal; Free Radical Biology and Medicine, Available online 6 April 2013.
http://dx.doi.org/10.1016/j.freeradbiomed.2013.03.018

Keywords: Parkinson’s disease, Alzheimer’s disease, Huntington’s disease, Amyotrophic lateral sclerosis, Charcot-Marie-Tooth disease, Friedreich’s ataxia, Neurodegenerative diseases, Mitochondrial dysfunction, Creatine, Co-Q10, PGC-1α, Sirtuins.

Moving Forward on Shifting Sands: Ethical Regulation of Gene Therapy Clinical Trials in the United Kingdom

Moving Forward on Shifting Sands: Ethical Regulation of Gene Therapy Clinical Trials in the United Kingdom. Emma Morris, Martin Gore, Andrew Baker and Adrian J Thrasher; Molecular Therapy (2013); 21 4, 715–716. doi:10.1038/mt.2013.43

Editorial. FULL TEXT

Gene Therapy Researchers' Assessments Of Risks And Perceptions Of Risk Acceptability In Clinical Trials

Gene Therapy Researchers' Assessments Of Risks And Perceptions Of Risk Acceptability In Clinical Trials. Claire T. Deakin, Ian E. Alexander, Cliff A. Hooker, Ian H. Kerridge; Molecular Therapy (2013); 21 4, 806–815. doi:10.1038/mt.2012.230

Decisions about clinical trials appear to be influenced not only by the clinical context and preclinical evidence. Identifying moral assumptions and qualitative assessments underpinning the design and conduct of research may facilitate future decision-making in clinical trials.