Santhera to Discontinue Sale of Catena® in Canada. 12:40 EST 27 Feb 2013 | Thomson Reuters.
Liestal, Switzerland, February 27, 2013 - Santhera Pharmaceuticals (SIX: SANN) announced today the voluntary withdrawal of Catena® from the Canadian market. This decision follows review of additional data from clinical trials in patients with Friedreich's Ataxia, and subsequent consultation with Health Canada.
Santera's web page
Wednesday, February 27, 2013
Monday, February 25, 2013
White House announces new US open access policy
White House announces new US open access policy. Newsblog (nature.com), 22 Feb 2013, Posted by Richard Van Noorden.
In a long-awaited leap forward for open access, the US government said today that publications from taxpayer-funded research should be made free to read after a year’s delay – expanding a policy which until now has only applied to biomedical science.
With both the US and Europe supporting delayed-access to publications, the UK government looks increasingly isolated in its preference for immediate open access. That policy is due to come in from 1 April, but the details are not yet clear. Communication around the policy was yesterday criticized as “unacceptable” by a House of Lords inquiry.
In a long-awaited leap forward for open access, the US government said today that publications from taxpayer-funded research should be made free to read after a year’s delay – expanding a policy which until now has only applied to biomedical science.
With both the US and Europe supporting delayed-access to publications, the UK government looks increasingly isolated in its preference for immediate open access. That policy is due to come in from 1 April, but the details are not yet clear. Communication around the policy was yesterday criticized as “unacceptable” by a House of Lords inquiry.
Sunday, February 24, 2013
Very late-onset Friedreich ataxia: later than life expectancy?
Very late-onset Friedreich ataxia: later than life expectancy?. Vincent Alvarez, Pierre Arnold, Thierry Kuntzer; Journal of Neurology February 2013. DOI:10.1007/s00415-013-6874-6
Saturday, February 23, 2013
Mitochondrial Transport, Metabolism and ROS Production in Disease Models
Mitochondrial Transport, Metabolism and ROS Production in Disease Models. Peter Hollenbeck, reearch talk in the Samuel Colella Lecture Series in Neurodegeneration at the Institute for Neurodegenerative Diseases of the University of Pittsburgh School of Medicine.
The talk covered studies from the Hollenbeck laboratory on the etiology of Friedreich ataxia and Parkinson’s disease. The work implicates defects in axonal transport, metabolism and mitochondrial turnover in the death of neurons.
The talk covered studies from the Hollenbeck laboratory on the etiology of Friedreich ataxia and Parkinson’s disease. The work implicates defects in axonal transport, metabolism and mitochondrial turnover in the death of neurons.
Thursday, February 21, 2013
Job opportunity. Study of the role of glial cells and neurons-glia interactions in Alzheimer disease and Friedreich ataxia
Job opportunity. Study of the role of glial cells and neurons-glia interactions in Alzheimer disease and Friedreich ataxia. UNIVERSITE PARIS
Identification of physiopathological mechanisms in human degenerative pathologies and new therapeutic compounds using Drosophila and mice. Study of the role of glial cells and neurons-glia interactions in Alzheimer disease and Friedreich ataxia using approaches from genetics to behavioral studies
Identification of physiopathological mechanisms in human degenerative pathologies and new therapeutic compounds using Drosophila and mice. Study of the role of glial cells and neurons-glia interactions in Alzheimer disease and Friedreich ataxia using approaches from genetics to behavioral studies
Tuesday, February 19, 2013
Discovery in HIV May Solve Efficiency Problems for Gene Therapy
Discovery in HIV May Solve Efficiency Problems for Gene Therapy. Case Western Reserve University (2013, February 14). Discovery in HIV may solve efficiency problems for gene therapy.
Journal Reference:
Mastooreh Chamanian, Katarzyna J. Purzycka, Paul T. Wille, Janice S. Ha, David McDonald, Yong Gao, Stuart F.J. Le Grice, Eric J. Arts. A cis-Acting Element in Retroviral Genomic RNA Links Gag-Pol Ribosomal Frameshifting to Selective Viral RNA Encapsidation. Cell Host & Microbe, 2013; 13 (2): 181 DOI: 10.1016/j.chom.2013.01.007
Journal Reference:
Mastooreh Chamanian, Katarzyna J. Purzycka, Paul T. Wille, Janice S. Ha, David McDonald, Yong Gao, Stuart F.J. Le Grice, Eric J. Arts. A cis-Acting Element in Retroviral Genomic RNA Links Gag-Pol Ribosomal Frameshifting to Selective Viral RNA Encapsidation. Cell Host & Microbe, 2013; 13 (2): 181 DOI: 10.1016/j.chom.2013.01.007
“Generación de Un Modelo Experimental Humano de Ataxia de Friedreich Mediante Células Troncales Pluripotentes Inducidas (Ipsc)”
“Generación de Un Modelo Experimental Humano de Ataxia de Friedreich Mediante Células Troncales Pluripotentes Inducidas (Ipsc)”. "Offer of employment"
El proyecto “Generación de Un Modelo Experimental Humano de Ataxia de Friedreich Mediante Células Troncales Pluripotentes Inducidas (Ipsc)” es un proyecto financiado a través de la convocatoria de la Consejería de Salud y Bienestar Social del año 2010, con número de expediente 726/2010.
El proyecto “Generación de Un Modelo Experimental Humano de Ataxia de Friedreich Mediante Células Troncales Pluripotentes Inducidas (Ipsc)” es un proyecto financiado a través de la convocatoria de la Consejería de Salud y Bienestar Social del año 2010, con número de expediente 726/2010.
Epigenetics in Friedreich's Ataxia: Challenges and Opportunities for Therapy
Epigenetics in Friedreich's Ataxia: Challenges and Opportunities for Therapy. Chiranjeevi Sandi, Sahar Al-Mahdawi, and Mark A. Pook. Genetics Research International, Volume 2013 (2013), Article ID 852080, 12 pages. http://dx.doi.org/10.1155/2013/852080
OPEN ACCESS
OPEN ACCESS
Thursday, February 14, 2013
Friedreich's Ataxia
Friedreich's Ataxia. P.E. Hart, A.H.V. Schapira, Encyclopedia of Biological Chemistry, 2013, Pages 332-334. http://dx.doi.org/10.1016/B978-0-12-378630-2.00443-6.
Keywords: Friedreich's ataxia (FRDA), ataxia, sensory loss, cardiomyopathy, skeletal abnormalities, diabetes, optic atrophy, frataxin, neurodegenerative disorder.
Keywords: Friedreich's ataxia (FRDA), ataxia, sensory loss, cardiomyopathy, skeletal abnormalities, diabetes, optic atrophy, frataxin, neurodegenerative disorder.
Pharmacological Screening Using an FXN-EGFP Cellular Genomic Reporter Assay for the Therapy of Friedreich Ataxia
Pharmacological Screening Using an FXN-EGFP Cellular Genomic Reporter Assay for the Therapy of Friedreich Ataxia. Lingli Li, Lucille Voullaire, Chiranjeevi Sandi, Mark A. Pook, Panos A. Ioannou, Martin B. Delatycki, Joseph P. Sarsero. PLoS ONE 8(2): e55940. doi:10.1371/journal.pone.0055940 (2013)
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Abstract
Friedreich ataxia (FRDA) is an autosomal recessive disorder characterized by neurodegeneration and cardiomyopathy. The presence of a GAA trinucleotide repeat expansion in the first intron of the FXN gene results in the inhibition of gene expression and an insufficiency of the mitochondrial protein frataxin. There is a correlation between expansion length, the amount of residual frataxin and the severity of disease. As the coding sequence is unaltered, pharmacological up-regulation of FXN expression may restore frataxin to therapeutic levels. To facilitate screening of compounds that modulate FXN expression in a physiologically relevant manner, we established a cellular genomic reporter assay consisting of a stable human cell line containing an FXN-EGFP fusion construct, in which the EGFP gene is fused in-frame with the entire normal human FXN gene present on a BAC clone. The cell line was used to establish a fluorometric cellular assay for use in high throughput screening (HTS) procedures. A small chemical library containing FDA-approved compounds and natural extracts was screened and analyzed. Compound hits identified by HTS were further evaluated by flow cytometry in the cellular genomic reporter assay. The effects on FXN mRNA and frataxin protein levels were measured in lymphoblast and fibroblast cell lines derived from individuals with FRDA and in a humanized GAA repeat expansion mouse model of FRDA. Compounds that were established to increase FXN gene expression and frataxin levels included several anti-cancer agents, the iron-chelator deferiprone and the phytoalexin resveratrol.
OPEN ACCESS
Abstract
Friedreich ataxia (FRDA) is an autosomal recessive disorder characterized by neurodegeneration and cardiomyopathy. The presence of a GAA trinucleotide repeat expansion in the first intron of the FXN gene results in the inhibition of gene expression and an insufficiency of the mitochondrial protein frataxin. There is a correlation between expansion length, the amount of residual frataxin and the severity of disease. As the coding sequence is unaltered, pharmacological up-regulation of FXN expression may restore frataxin to therapeutic levels. To facilitate screening of compounds that modulate FXN expression in a physiologically relevant manner, we established a cellular genomic reporter assay consisting of a stable human cell line containing an FXN-EGFP fusion construct, in which the EGFP gene is fused in-frame with the entire normal human FXN gene present on a BAC clone. The cell line was used to establish a fluorometric cellular assay for use in high throughput screening (HTS) procedures. A small chemical library containing FDA-approved compounds and natural extracts was screened and analyzed. Compound hits identified by HTS were further evaluated by flow cytometry in the cellular genomic reporter assay. The effects on FXN mRNA and frataxin protein levels were measured in lymphoblast and fibroblast cell lines derived from individuals with FRDA and in a humanized GAA repeat expansion mouse model of FRDA. Compounds that were established to increase FXN gene expression and frataxin levels included several anti-cancer agents, the iron-chelator deferiprone and the phytoalexin resveratrol.
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