Cargando…

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

TALEs targeting a promoter sequence and fused with a transcription activation domain (TAD) may be used to specifically induce the expression of a gene as a potential treatment for haploinsufficiency. This potential therapeutic approach was applied to increase the expression of frataxin in fibroblast...

Descripción completa

Detalles Bibliográficos
Autores principales:	Chapdelaine, Pierre, Coulombe, Zoé, Chikh, Amina, Gérard, Catherine, Tremblay, Jacques P
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	Nature Publishing Group 2013
Materias:	Original Article
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4028015/ https://www.ncbi.nlm.nih.gov/pubmed/24002729 http://dx.doi.org/10.1038/mtna.2013.41

Ejemplares similares

An AAV9 coding for frataxin clearly improved the symptoms and prolonged the life of Friedreich ataxia mouse models
por: Gérard, Catherine, et al.
Publicado: (2014)

Increased Frataxin Expression Induced in Friedreich Ataxia Cells by Platinum TALE-VP64s or Platinum TALE-SunTag
por: Cherif, Khadija, et al.
Publicado: (2018)

Frataxin levels in peripheral tissue in Friedreich ataxia
por: Lazaropoulos, Michael, et al.
Publicado: (2015)

Posttranslational regulation of mitochondrial frataxin and identification of compounds that increase frataxin levels in Friedreich’s ataxia
por: Hackett, Peter T., et al.
Publicado: (2022)

Neurodegeneration in Friedreich's Ataxia: From Defective Frataxin to Oxidative Stress
por: Gomes, Cláudio M., et al.
Publicado: (2013)

Selected missense mutations impair frataxin processing in Friedreich ataxia
Publicado: (2017)

Selected missense mutations impair frataxin processing in Friedreich ataxia
por: Clark, Elisia, et al.
Publicado: (2017)

Simultaneous Quantification of Mitochondrial Mature Frataxin and Extra-Mitochondrial Frataxin Isoform E in Friedreich’s Ataxia Blood
por: Wang, Qingqing, et al.
Publicado: (2022)

Iron-dependent regulation of frataxin expression: implications for treatment of Friedreich ataxia
por: Li, Kuanyu, et al.
Publicado: (2008)

Novel Frataxin Isoforms May Contribute to the Pathological Mechanism of Friedreich Ataxia
por: Xia, Haiyan, et al.
Publicado: (2012)

Novel Point Mutations in Frataxin Gene in Iranian Patients with Friedreich’s Ataxia
por: HEIDARI, Mohammad Mehdi, et al.
Publicado: (2014)

DNA methylation in Friedreich ataxia silences expression of frataxin isoform E
por: Rodden, Layne N., et al.
Publicado: (2022)

HDAC Inhibitors Correct Frataxin Deficiency in a Friedreich Ataxia Mouse Model
por: Rai, Myriam, et al.
Publicado: (2008)

Development of Frataxin Gene Expression Measures for the Evaluation of Experimental Treatments in Friedreich’s Ataxia
por: Plasterer, Heather L., et al.
Publicado: (2013)

Extra-mitochondrial mouse frataxin and its implications for mouse models of Friedreich’s ataxia
por: Weng, Liwei, et al.
Publicado: (2020)

Limitations in a frataxin knockdown cell model for Friedreich ataxia in a high-throughput drug screen
por: Calmels, Nadège, et al.
Publicado: (2009)

Friedreich's Ataxia, Frataxin, PIP5K1B: Echo of a Distant Fracas
por: Bayot, Aurélien, et al.
Publicado: (2013)

Dimethyl fumarate dosing in humans increases frataxin expression: A potential therapy for Friedreich’s Ataxia
por: Jasoliya, Mittal, et al.
Publicado: (2019)

Interferon gamma upregulates frataxin and corrects the functional deficits in a Friedreich ataxia model
por: Tomassini, Barbara, et al.
Publicado: (2012)

The Friedreich's ataxia protein frataxin modulates DNA base excision repair in prokaryotes and mammals
por: Thierbach, René, et al.
Publicado: (2010)

Frataxin deficiency increases cyclooxygenase 2 and prostaglandins in cell and animal models of Friedreich's ataxia
por: Hayashi, Genki, et al.
Publicado: (2014)

Dyclonine rescues frataxin deficiency in animal models and buccal cells of patients with Friedreich's ataxia
por: Sahdeo, Sunil, et al.
Publicado: (2014)

Frataxin deficiency alters gene expression in Friedreich ataxia derived IPSC‐neurons and cardiomyocytes
por: Angulo, Mariana B., et al.
Publicado: (2022)

Repeat-induced epigenetic changes in intron 1 of the frataxin gene and its consequences in Friedreich ataxia
por: Greene, Eriko, et al.
Publicado: (2007)

Low apolipoprotein A-I levels in Friedreich’s ataxia and in frataxin-deficient cells: Implications for therapy
por: Wang, QingQing, et al.
Publicado: (2018)

Frataxin‐deficient neurons and mice models of Friedreich ataxia are improved by TAT‐MTScs‐FXN treatment
por: Britti, Elena, et al.
Publicado: (2017)

Role of frataxin protein deficiency and metabolic dysfunction in Friedreich ataxia, an autosomal recessive mitochondrial disease
por: Clark, Elisia, et al.
Publicado: (2018)

Recent Advances in the Elucidation of Frataxin Biochemical Function Open Novel Perspectives for the Treatment of Friedreich’s Ataxia
por: Monfort, Beata, et al.
Publicado: (2022)

Intrathecal delivery of frataxin mRNA encapsulated in lipid nanoparticles to dorsal root ganglia as a potential therapeutic for Friedreich’s ataxia
por: Nabhan, Joseph F., et al.
Publicado: (2016)

Nrf2-Inducers Counteract Neurodegeneration in Frataxin-Silenced Motor Neurons: Disclosing New Therapeutic Targets for Friedreich’s Ataxia
por: Petrillo, Sara, et al.
Publicado: (2017)

The First Cellular Models Based on Frataxin Missense Mutations That Reproduce Spontaneously the Defects Associated with Friedreich Ataxia
por: Calmels, Nadège, et al.
Publicado: (2009)

Time-resolved functional analysis of acute impairment of frataxin expression in an inducible cell model of Friedreich ataxia
por: Poburski, Dörte, et al.
Publicado: (2016)

Peptide SS-31 upregulates frataxin expression and improves the quality of mitochondria: implications in the treatment of Friedreich ataxia
por: Zhao, Hongting, et al.
Publicado: (2017)

E3 Ligase RNF126 Directly Ubiquitinates Frataxin, Promoting Its Degradation: Identification of a Potential Therapeutic Target for Friedreich Ataxia
por: Benini, Monica, et al.
Publicado: (2017)

Mesenchymal Stem Cells Restore Frataxin Expression and Increase Hydrogen Peroxide Scavenging Enzymes in Friedreich Ataxia Fibroblasts
por: Kemp, Kevin, et al.
Publicado: (2011)

Lentivirus-meditated frataxin gene delivery reverses genome instability in Friedreich ataxia patient and mouse model fibroblasts
por: Khonsari, H, et al.
Publicado: (2016)

Adding a temporal dimension to the study of Friedreich's ataxia: the effect of frataxin overexpression in a human cell model
por: Vannocci, Tommaso, et al.
Publicado: (2018)

Frataxin gene editing rescues Friedreich’s ataxia pathology in dorsal root ganglia organoid-derived sensory neurons
por: Mazzara, Pietro Giuseppe, et al.
Publicado: (2020)

SINEUP non-coding RNAs rescue defective frataxin expression and activity in a cellular model of Friedreich's Ataxia
por: Bon, Carlotta, et al.
Publicado: (2019)

Liquid Chromatography–Mass Spectrometry Analysis of Frataxin Proteoforms in Whole Blood as Biomarkers of the Genetic Disease Friedreich’s Ataxia
por: Rojsajjakul, Teerapat, et al.
Publicado: (2023)

Cannot write session to /tmp/vufind_sessions/sess_ngbik41qh9phk39ntsq645qqbv