Tuesday, August 31, 2010

Symposium participants optimistic about finding first treatment for Friedreich's ataxia

USF Health News, August 30, 2010 @ 4:58 pm

"With all the significant scientific advancements presented, at the end of the symposium it was the people with Friedreich’s ataxia who gave the research meaning and value"

Saturday, August 28, 2010

Proteomic Analysis of Protein-Protein Interactions within the CSD Fe-S Cluster Biogenesis System

J. Proteome Res., Just Accepted Manuscript, Publication Date (Web): August 24, 2010

Heather May Bolstad , Danielle J Botelho and Matthew James Wood

Keygen: Fe-S cluster biogenesis, cysteine desulfurase CsdA, sulfur acceptor protein CsdE, E1-like protein CsdL. Fe-S cluster assembly (ErpA, glutaredoxin-3, glutaredoxin-4), sulfur trafficking (CsdL, YchN) proteins, two-pathway model.

FULL TEXY PDF

Friday, August 27, 2010

Lipid Peroxides: More Sophisticated Than Their Reputation

ScienceDaily (Aug. 24, 2010) — Accumulation of lipid peroxides in the cell are associated with diseases and cellular stress. In the current issue of Proceedings of the National Academy of Sciences, researchers at Helmholtz Zentrum München and the Swedish medical university Karolinska Institutet show that lipid peroxides also play an important, yet-unrecognized role in the regulation of receptor tyrosine kinases.

Reference scientific paper:
12/15-lipoxygenase-derived lipid peroxides control receptor tyrosine kinase signaling through oxidation of protein tyrosine phosphatases.
M. Conrad, A. Sandin, H. Forster, A. Seiler, J. Frijhoff, M. Dagnell, G. W. Bornkamm, O. Radmark, R. Hooft van Huijsduijnen, P. Aspenstrom, F. Bohmer, A. Ostman. Proceedings of the National Academy of Sciences, 2010; DOI: 10.1073/pnas.1007909107

Wednesday, August 25, 2010

The Monash University are seeking healthy participants for the control group, Understanding Motor Deficits in Friedreich's Ataxia

The aim of this study is to investigate the extent of motor overflow in people with Friedreich's ataxia. Motor overflow refers to involuntary movement which occurs on the opposite side of the body when voluntary movement takes place on one side.

Defects in Mitochondrial Axonal Transport and Membrane Potential without Increased Reactive Oxygen Species Production in a Drosophila Model of Friedreich Ataxia

The Journal of Neuroscience, August 25, 2010, 30(34):11369-11378; doi:10.1523/JNEUROSCI.0529-10.2010

Yujiro Shidara {dagger} and Peter J. Hollenbeck
Department of Biological Sciences, Purdue University, West Lafayette, Indiana 47907

KEYWORDS: Friedreich ataxia, frataxin deficiency, oxidative stress, cellular neuropathology, mitochondrial axonal transport, membrane potential (MMP), reactive oxygen species (ROS), neuromuscular junctions (NMJs), antimycin A.

Long-term effects of coordinative training in degenerative cerebellar disease†

Movement Disorders, Article first published online: 24 AUG 2010, DOI: 10.1002/mds.23222

Winfried Ilg PhD1, Doris Brötz PT2, 3. Susanne Burkard PT3, Martin A. Giese PhD1, Ludger Schöls MD4,*, Matthis Synofzik MD4.

Tuesday, August 24, 2010

Prospects for the Use of Artificial Chromosomes and Minichromosome-Like Episomes in Gene Therapy

Journal of Biomedicine and Biotechnology, Volume 2010 (2010), Article ID 642804, 16 pages, doi:10.1155/2010/642804

Sara Pérez-Luz1,2,3 and Javier Díaz-Nido1,2,3

1Departamento de Biología Molecular, Universidad Autónoma de Madrid, 28049 Madrid, Spain
2Centro de Biología Molecular “Severo Ochoa” (CSIC-UAM), C/Nicolás Cabrera 1, Universidad Autónoma de Madrid, 28049 Madrid, Spain
3U-748, Area de Neurogenética, Centro de Investigación Biomédica en Red de Enfermedades Raras (CIBERER), Spain

OPEN ACCESS

Abstract

Artificial chromosomes and minichromosome-like episomes are large DNA molecules capable of containing whole genomic loci, and be maintained as nonintegrating, replicating molecules in proliferating human somatic cells. Authentic human artificial chromosomes are very difficult to engineer because of the difficulties associated with centromere structure, so they are not widely used for gene-therapy applications. However, OriP/EBNA1-based episomes, which they lack true centromeres, can be maintained stably in dividing cells as they bind to mitotic chromosomes and segregate into daughter cells. These episomes are more easily engineered than true human artificial chromosomes and can carry entire genes along with all their regulatory sequences. Thus, these constructs may facilitate the long-term persistence and physiological regulation of the expression of therapeutic genes, which is crucial for some gene therapy applications. In particular, they are promising vectors for gene therapy in inherited diseases that are caused by recessive mutations, for example haemophilia A and Friedreich's ataxia. Interestingly, the episome carrying the frataxin gene (deficient in Friedreich's ataxia) has been demonstrated to rescue the susceptibility to oxidative stress which is typical of fibroblasts from Friedreich's ataxia patients. This provides evidence of their potential to treat genetic diseases linked to recessive mutations through gene therapy.

Section: "4.4. Gene Therapy for Friedreich’s Ataxia"

FULLTEXT PDF

Mitochondrial protein import: from proteomics to functional mechanisms

Nature Reviews Molecular Cell Biology 11, 655–667 (1 September 2010) | doi:10.1038/nrm2959

Oliver Schmidt , Nikolaus Pfanner & Chris Meisinger

Keywords: Mitochondria, proteins, cytosol, import pathways, protein translocases.

Sunday, August 22, 2010

Cytosolic Iron-Sulfur Cluster Assembly (CIA) System: Factors, Mechanism, and Relevance to Cellular Iron Regulation

J. Biol. Chem. 2010 285: 26745-26751. doi:10.1074/jbc.R110.122218

Anil K Sharma, Leif J Pallesen, Robert J Spang and William E Walden*

University of Illinois at Chicago, United States

KEYWORDS: Iron sulfur (FeS) cluster biogenesis, cellular iron homeostasis.

Friday, August 20, 2010

Expression of Human Frataxin Is Regulated by Transcription Factors SRF and TFAP2

Li K, Singh A, Crooks DR, Dai X, Cong Z, et al. 2010 . PLoS ONE 5(8): e12286. doi:10.1371/journal.pone.0012286

OPEN ACCESS

Abstract
Background

Friedreich ataxia is an autosomal recessive neurodegenerative disease caused by reduced expression levels of the frataxin gene (FXN) due to expansion of triplet nucleotide GAA repeats in the first intron of FXN. Augmentation of frataxin expression levels in affected Friedreich ataxia patient tissues might substantially slow disease progression.
Methodology/Principal Findings

We utilized bioinformatic tools in conjunction with chromatin immunoprecipitation and electrophoretic mobility shift assays to identify transcription factors that influence transcription of the FXN gene. We found that the transcription factors SRF and TFAP2 bind directly to FXN promoter sequences. SRF and TFAP2 binding sequences in the FXN promoter enhanced transcription from luciferase constructs, while mutagenesis of the predicted SRF or TFAP2 binding sites significantly decreased FXN promoter activity. Further analysis demonstrated that robust SRF- and TFAP2-mediated transcriptional activity was dependent on a regulatory element, located immediately downstream of the first FXN exon. Finally, over-expression of either SRF or TFAP2 significantly increased frataxin mRNA and protein levels in HEK293 cells, and frataxin mRNA levels were also elevated in SH-SY5Y cells and in Friedreich ataxia patient lymphoblasts transfected with SRF or TFAP2.
Conclusions/Significance

We identified two transcription factors, SRF and TFAP2, as well as an intronic element encompassing EGR3-like sequence, that work together to regulate expression of the FXN gene. By providing new mechanistic insights into the molecular factors influencing frataxin expression, our results should aid in the discovery of new therapeutic targets for the treatment of Friedreich ataxia.

FULL TEXT PDF