Limitations in a frataxin knockdown cell model for Friedreich ataxia in a high-throughput drug screen
H. Seznec
BMC Neurology, 2009
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Activation of Frataxin Protein Expression by Antisense Oligonucleotides Targeting the Mutant Expanded Repeat
Vivek Sharma
Nucleic acid therapeutics, 2018
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Adding a temporal dimension to the study of Friedreich's ataxia: the effect of frataxin overexpression in a human cell model
Luca Quaroni
Disease models & mechanisms, 2018
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The First Cellular Models Based on Frataxin Missense Mutations That Reproduce Spontaneously the Defects Associated with Friedreich Ataxia
Marie Wattenhofer-Donzé
PLoS ONE, 2009
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Selection of Synthetic Proteins to Modulate the Human Frataxin Function
Martin Aran
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Increasing frataxin gene expression with histone deacetylase inhibitors as a therapeutic approach for Friedreich's ataxia
Joel Gottesfeld
Journal of Neurochemistry, 2013
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An AAV9 coding for frataxin clearly improved the symptoms and prolonged the life of Friedreich ataxia mouse models
Mohammed Filali
Molecular Therapy — Methods & Clinical Development, 2014
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Functional recovery in a Friedreich's ataxia mouse model by frataxin gene transfer using an HSV-1 amplicon vector
Javier Diaz-Nido
Molecular …, 2007
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Cell and Gene Therapy for Friedreich Ataxia: Progress to Date
Marguerite Evans-Galea
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Deletion of the GAA repeats from the human frataxin gene using the CRISPR-Cas9 system in YG8R-derived cells and mouse models of Friedreich ataxia
khadija Cherif
Gene Therapy, 2016
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Functional genomic analysis of frataxin deficiency reveals tissue-specific alterations and identifies the PPAR pathway as a therapeutic target in Friedreich's ataxia
Giovanni Coppola
Human Molecular Genetics, 2009
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Beyond Loss of Frataxin: the Complex Molecular Pathology of Friedreich Ataxia
Marguerite Evans-Galea
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Frataxin-deficient neurons and mice models of Friedreich ataxia are improved by TAT-MTScs-FXN treatment
elena britti
Journal of cellular and molecular medicine, 2017
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Neurobehavioral deficits of mice expressing a low level of G127V mutant frataxin
Aamir Zuberi
Neurobiology of Disease
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Preventing the ubiquitin-proteasome-dependent degradation of frataxin, the protein defective in Friedreich's ataxia
Natascia Ventura
Human Molecular Genetics, 2011
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High levels of frataxin overexpression leads to mitochondrial and cardiac toxicity in mouse models
Laurent Monassier
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Increased Frataxin Expression Induced in Friedreich Ataxia Cells by Platinum TALE-VP64s or Platinum TALE-SunTag
khadija Cherif
Molecular Therapy - Nucleic Acids, 2018
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Gene expression profiling in frataxin deficient mice: Microarray evidence for significant expression changes without detectable neurodegeneration
Giovanni Coppola
Neurobiology of Disease, 2006
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HDAC Inhibitors Correct Frataxin Deficiency in a Friedreich Ataxia Mouse Model
Kiki April
PLOS One, 2008
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Functional genomic analysis of frataxin deficiency reveals tissue-specific alterations and identifies the PPARγ pathway as a therapeutic target in Friedreich's ataxia
Sergio Cocozza
Human molecular …, 2009
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Friedreich Ataxia: From GAA Triplet–Repeat Expansion to Frataxin Deficiency
Pragna Patel
The American Journal of Human Genetics, 2001
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Biophysical characterisation of the recombinant human frataxin precursor
Alejandro Ferrari
FEBS open bio, 2018
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Reduction in frataxin causes progressive accumulation of mitochondrial damage
Grazia Isaya
Human Molecular Genetics, 2003
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Exenatide induces frataxin expression and improves mitochondrial function in Friedreich ataxia
Jean-christophe Jonas
JCI Insight
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Synthesis of frataxin genes by direct assembly of serial deoxyoligonucleotide primers and its expression in Escherichia coli
Young Geol Yoon
Biotechnology and Bioprocess Engineering, 2013
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A new cellular model to follow Friedreich's ataxia development in a time-resolved way
Salvatore Adinolfi
Disease Models & Mechanisms, 2015
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Infectious delivery and expression of a 135 kb human FRDA genomic DNA locus complements Friedreich's ataxia deficiency in human cells
Javier Diaz-Nido
Molecular …, 2007
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Reduction of Caenorhabditis elegans frataxin increases sensitivity to oxidative stress, reduces lifespan, and causes lethality in a mitochondrial complex II mutant
Pilar Cabo
The FASEB Journal, 2005
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Frataxin Silencing Inactivates Mitochondrial Complex I in NSC34 Motoneuronal Cells and Alters Glutathione Homeostasis
Anna Pastore
International Journal of Molecular Sciences, 2014
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