Gene Therapy Emerges From Disgrace to Be the Next Big Thing, Again

Gene Therapy Emerges From Disgrace to Be the Next Big Thing, Again. By Carl Zimmer 08.13.13. Wired Science wired.com. Wired Science Blogs/Genetic future

The complex world of proteins: Structure, function, and oligomerization of frataxin

The complex world of proteins: Structure, function, and oligomerization of frataxin. Christopher Söderberg; University dissertation from Department of Chemistry, Lund University (2013-09-27)

Keywords: Friedreich's ataxia, Frataxin, self-assembly, protein-protein interaction, iron homeostasis, SAXS, cross-linking.

Full text is not available.

The legal imperative for treating rare disorders

The legal imperative for treating rare disorders. Hanna I Hyry, Jonathan CP Roos, Jeremy Manuel and Timothy M Cox; Orphanet Journal of Rare Diseases 2013, 8:135 doi:10.1186/1750-1172-8-135

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Mitochondrial iron transport and homeostasis in plants

Mitochondrial iron transport and homeostasis in plants. Anshika Jain and Erin L. Connolly. Front. Plant Sci., 06 September 2013 | doi: 10.3389/fpls.2013.00348

"Frataxin functions in plants"

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How to Design a Cell or Gene Therapy Clinical Trial: Advice from the FDA

How to Design a Cell or Gene Therapy Clinical Trial: Advice from the FDA. Adrian P. Gee; Molecular Therapy (2013); 21 9, 1639–1640. doi:10.1038/mt.2013.172

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Friedreich's ataxia research leads to a new understanding of the disease

Friedreich's ataxia research leads to a new understanding of the disease .

06/09/2013. Researchers at the University of Minnesota are using magnetic resonance spectroscopy to better understand how Friedreich's ataxia affects both the brain and spinal cord.

Ataxie de Friedreich : essai ACTFRIE

Ataxie de Friedreich : essai ACTFRIE.

L'objectif de l'essai ACTFRIE, soutenu financièrement par l'AFM-Téléthon, est d'évaluer l'efficacité de la pioglitazone comparativement à un placebo sur l'atteinte neurologique des personnes atteintes de maladie de Friedreich.

Les 40 participants prévus pour cet essai sont inclus. L'essai, qui a démarré en décembre 2008, est toujours en cours. Sa fin est prévue en avril 2014 et les premiers résultats pourraient être disponibles fin 2014.

A mitochondrial implication in a Tunisian patient with Friedreich's ataxia-like

A mitochondrial implication in a Tunisian patient with Friedreich's ataxia-like. Une implication mitochondriale chez un patient tunisien atteint d’une ataxie de Friedreich-like. M. Maalej, E. Mkaouar-Rebai, M. Mnif, N. Mezghani, I. Ben Ayed, I. Chamkha, M. Abid, F. Fakhfakh; Pathologie Biologie, Available online 4 September 2013

Friedreich Ataxia Patient Tissues Exhibit Increased 5-Hydroxymethylcytosine Modification and Decreased CTCF Binding at the FXN Locus

Friedreich Ataxia Patient Tissues Exhibit Increased 5-Hydroxymethylcytosine Modification and Decreased CTCF Binding at the FXN Locus . Sahar Al-Mahdawi, Chiranjeevi Sandi, Ricardo Mouro Pinto, Mark A. Pook; PLoS ONE 8(9): e74956. doi:10.1371/journal.pone.0074956


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A Potential New Therapeutic Approach for Friedreich Ataxia: Induction of Frataxin Expression With TALE Proteins

A Potential New Therapeutic Approach for Friedreich Ataxia: Induction of Frataxin Expression With TALE Proteins. Pierre Chapdelaine, Zoé Coulombe, Amina Chikh, Catherine Gérard and Jacques P Tremblay. Molecular Therapy Nucleic Acids (2013) 2, e119; doi:10.1038/mtna.2013.41
Published online 3 September 2013.

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