A 'Disruptive Science' Ready For Commercial Development - Gene Therapy

Mary Ann Liebert, Inc./Genetic Engineering News. (2012, January 26). "A 'Disruptive Science' Ready For Commercial Development - Gene Therapy." Medical News Today.

"The time for commercial development of gene therapy has come. Patients with diseases treatable and curable with gene therapy deserve access to the technology, which has demonstrated both its effectiveness and feasibility"

The promise of induced pluripotent stem cells in research and therapy

Nature 481, 295–305 (19 January 2012), doi:10.1038/nature10761

Daisy A. Robinton & George Q. Daley

Keywords: stem-cell biology, reprogramming technology, pluripotency, somatic cells, personalized regenerative cell therapies.

Supplementary Table (128K)

The mismatch repair system protects against intergenerational GAA repeat instability in a Friedreich ataxia mouse model

Neurobiology of Disease, Available online 20 January 2012, http://dx.doi.org/10.1016/j.nbd.2012.01.002

Vahid Ezzatizadeh a,Ricardo Mouro Pinto b, Chiranjeevi Sandi a, Madhavi Sandi a, Sahar Al-Mahdawi a, Hein te Riele c, Mark A. Pook a.
a Division of Biosciences, School of Health Sciences and Social Care, Brunel University, Uxbridge, UB8 3PH, UK
b Molecular Neurogenetics Unit, Center for Human Genetic Research, Massachusetts General Hospital, Boston, Massachusetts, USA
c Division of Molecular Biology, The Netherlands Cancer Institute, Plesmanlaan 121, 1066 CX Amsterdam, The Netherlands

Keywords: Friedreich ataxia (FRDA), GAA repeat expansion mutation, FXN gene, mismatch repair (MMR) proteins, TNR instability, Msh2, Msh3, Msh6, Pms2,

Oxidative stress induces mitochondrial fragmentation in frataxin-deficient cells

Biochemical and Biophysical Research Communications, Available online 16 January 2012, In Press, Accepted Manuscript. http://dx.doi.org/10.1016/j.bbrc.2012.01.022

Sophie Lefevre a, b, 1, Dominika Sliwa a, 1, Pierre Rustin c, d, Jean-Michel Camadro a, Renata Santos a.
a Mitochondria, Metals and Oxidative Stress Laboratory, Institut Jacques Monod, CNRS-Université Paris-Diderot, Sorbonne Paris Cité, 15 rue Hélène Brion, 75205 Paris cedex 13, France
b ED515 UPMC, 4 place Jussieu 75005 Paris, France
c Inserm, U676, Physiopathology and Therapy of Mitochondrial Disease Laboratory, 75019 Paris, France
d Université Paris-Diderot, Faculté de Médecine Denis Diderot, IFR02, Paris, France

Keywords: Friedreich ataxia (FA), mitochondrial frataxin, iron-sulfur cluster assembly, fragmented mitochondria, oxidative stress, mitochondrial dynamics.

Hippotherapy: An Intervention to Habilitate Balance Deficits in Children With Movement Disorders--A Clinical Trial.

Phys Ther. 2012 Jan 12, Published online before print, doi: 10.2522/​ptj.20110081

Silkwood-Sherer DJ, Killian CB, Long TM, Martin KS.

D.J Silkwood-Sherer, PT, DHS, HPCS, Graduate Program in Physical Therapy, Herbert H. and Grace A. Dow College of Health Professions, 1202 Health Professions Bldg, Central Michigan University, Mt Pleasant, MI 48859 (USA).

Keywords: hippotherapy, children with movement disorders, postural instability, mild to moderate balance problems, Activities Scale for Kids-Performance (ASKp), daily life skills.


Hippotherapy may be a viable treatment strategy for children with mild to moderate balance problems to improve balance deficits and increase performance of daily life skills.

Effects of Friedreich's ataxia GAA repeats on DNA replication in mammalian cells

Nucl. Acids Res. (2012) doi: 10.1093/nar/gks021.

Gurangad S. Chandok 1, Mayank P. Patel 1, Sergei M. Mirkin 2 and Maria M. Krasilnikova
Department of Biochemistry and Molecular Biology, Penn State University, University Park, PA 16802 and 2Department of Biology, Tufts University.

Keyword: Friedreich's ataxia (FRDA),(GAA)n repeat, age-dependent instability, episomal DNA replication, chromatin.

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Reactive Oxygen Species and Aging in Caenorhabditis elegans: Causal or Casual Relationship?

Jeremy Michael Van Raamsdonk and Siegfried Hekimi and. Antioxidants & Redox Signaling. December 15, 2010, 13(12): 1911-1953. doi:10.1089/ars.2010.3215.

Keywords: reactive oxygen species (ROS), aging, Caenorhabditis elegans, frh-1-frataxin

S-Glutathionylation: From Molecular Mechanisms to Health Outcomes

Antioxidants & Redox Signaling. July 1, 2011, 15(1): 233-270. doi:10.1089/ars.2010.3540.

Ying Xiong, Joachim D. Uys, Kenneth D. Tew, and Danyelle M. Townsend.

KEYWORDS: Redox homeostasis, human disease pathologies, glutathione, Friedreich's ataxia

Mechanisms of Altered Redox Regulation in Neurodegenerative Diseases—Focus on S-Glutathionylation

Antioxidants & Redox Signaling. null, Vol. 0, No. 0,-Not available-, ahead of print. doi:10.1089/ars.2011.4119.

Elizabeth A. Sabens Liedhegner 1, Xing-Huang Gao 2, and John J. Mieyal 2,3
1Neuroscience Research Center, Medical College of Wisconsin, Milwaukee, Wisconsin.
2Department of Pharmacology, Case Western Reserve University, School of Medicine, Cleveland, Ohio.
3Louis B. Stokes Veterans Affairs Medical Research Center, Cleveland, Ohio.



Keywords: Neurodegenerative diseases, oxidative stress, reactive oxygen species (ROS), reactive nitrogen species (RNS), glutathione (GSH), Friedreich's ataxia

The European Hospital Exemption Clause—New Option for Gene Therapy?

Human Gene Therapy.
Christian J. Buchholz, Ralf Sanzenbacher, and Silke Schüle. January 2012, 23(1): 7-12. doi:10.1089/hum.2011.2529.

Keywords: Gene-therapy medicinal products, authorized clinical trials, centralized procedure, European Medicines Agency, Regulation (EC) No. 1394/2007, cell-therapy products, hospital exemption, exclusive professional responsibility.