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FARA Cure FA

Functional Characterization of Friedreich Ataxia iPS-Derived Neuronal Progenitors and Their Integration in the Adult Brain

Friedreich ataxia (FRDA) is an autosomal recessive disease characterised by neurodegeneration and cardiomyopathy that is caused by an insufficiency of the mitochondrial protein, frataxin. Our previous studies described the generation of FRDA induced pluripotent stem cell lines (FA3 and FA4 iPS) that retained genetic characteristics of this disease.

Read More: Functional Characterization of Friedreich Ataxia iPS-Derived Neuronal Progenitors and Their Integration in the Adult Brain

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2020 (13)
- January (13)
  - • An Instrumented Measurement S…
  - • Predictors of loss of ambulat…
  - • Frataxin Structure and Functi…
  - • Safety and Efficacy of Interf…
  - • Frataxin deficiency in Friedr…
  - • Health related quality of lif…
  - • Multiple mechanisms underpin …
  - • Large-scale contractions of F…
  - • MDA Awards Venture Philanthro…
  - • Mammalian iron-sulfur cluster…
  - • Retrotope Expands its Drug Pi…
  - • Longitudinal Increases in Cer…
  - • Genetic Analysis of Hereditar…
2019 (102)
- December (16)
  - • Exenatide induces frataxin ex…
  - • Nanoscopic X-ray imaging and …
  - • Age of onset determines intri…
  - • A new tool to determine the c…
  - • Electrophysiological evidence…
  - • Neurochemical profiles in her…
  - • Iron Hack - A symposium/hacka…
  - • Chondrial Therapeutics Announ…
  - • Zafgen and Chondrial Therapeu…
  - • Exicure Announces First Neuro…
  - • Effects of tocotrienol supple…
  - • Friedreich's Ataxia: Case ser…
  - • Mitochondrial dysfunction in …
  - • Pediatric Neuromuscular Disor…
  - • N-Terminomics/TAILS Profiling…
  - • Possible Mechanisms of Biolog…
- November (11)
  - • Left ventricular structural a…
  - • Global Ataxia Advocacy Organi…
  - • Orphan Drugs In Development F…
  - • Temporal but not spatial dysm…
  - • Quantitative Proteomic and Ne…
  - • Refinement of protein Fe(II) …
  - • Evolutionarily conserved susc…
  - • Speech and Language Disorders…
  - • The Structure of the Human AC…
  - • Potential biomarker identific…
  - • The first biallelic missense …
- October (11)
  - • Targeting NRF2 for the Treatm…
  - • Cerebellum and cognition in F…
  - • Predictors of Left Ventricula…
  - • Induced pluripotent stem cell…
  - • Minoryx Therapeutics receives…
  - • Reata Announces Positive Topl…
  - • Primary Cultures of Pure Embr…
  - • Changes detected in swallowin…
  - • SINEUP non-coding RNAs rescue…
  - • Minoryx Therapeutics complete…
  - • Health-related quality of lif…
- September (6)
  - • Unique roles of iron and zinc…
  - • Mechanism of activation of th…
  - • Two new Pfizer-coauthored stu…
  - • Scoliosis in Patients With Fr…
  - • Friedreich ataxia- pathogenes…
  - • The Working Life of People wi…
- August (7)
  - • Backbone resonance assignment…
  - • Application of Quantitative M…
  - • Oral mobility reflects rate o…
  - • Excision of the expanded GAA …
  - • Folding and Dynamics are Stro…
  - • Nrf2 Induction Re-establishes…
  - • Physiologically relevant reco…
- July (8)
  - • Exploring iron-binding to hum…
  - • Low frataxin mRNA expression …
  - • REGENXBIO Announces New Licen…
  - • Molecular Mechanisms and Ther…
  - • New developments in pharmacot…
  - • The current state of biomarke…
  - • Correlation between frataxin …
  - • Evaluation of antibodies for …
- June (11)
  - • Frequency and Genetic Profile…
  - • FARA Job Posting: Associate D…
  - • Evidence for genetically dete…
  - • Open label Pilot Study of Ora…
  - • Deuterated Polyunsaturated Fa…
  - • Altered neocortical tactile b…
  - • Young Investigators: Call for…
  - • Microvascular pathology in Fr…
  - • p53 Binds Preferentially to N…
  - • Dimethyl fumarate dosing in h…
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  - • StrideBio and Takeda Sign Col…
  - • Patient reported outcomes in …
  - • Assessment of cell-free level…
  - • Inherited Ataxia and Intrathe…
  - • The role of robotic gait trai…
  - • The Role of Iron in Friedreic…
  - • Progress in understanding Fri…
- February (8)
  - • C-Path and FARA announce the …
  - • Minoryx Therapeutics receives…
  - • Guidelines on the diagnosis a…
  - • In Vitro interaction between …
  - • The role of mitochondrial lab…
  - • Pattern of Cerebellar Atrophy…
  - • Phosphodiesterase Inhibitors …
  - • Heart disease in Friedreich's…
- January (7)
  - • Neurocrine Biosciences and Vo…
  - • OGTT is recommended for gluco…
  - • Ferroptosis as a novel therap…
  - • Structural and functional cha…
  - • Longitudinal evaluation of ir…
  - • Drug repositioning screening …
  - • Differences in the determinan…
2018 (68)
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- November (7)
  - • FAST-1 antisense RNA epigenet…
  - • Characterization of a new N-t…
  - • Rapid and Complete Reversal o…
  - • Oligonucleotides Hold Promise…
  - • Voyager Therapeutics Announce…
  - • Safety, pharmacodynamics, and…
  - • Plasma Markers of Neurodegene…
- October (6)
  - • Intraepidermal Nerve Fiber De…
  - • Calcium Deregulation: Novel I…
  - • Developmental and Neurodegene…
  - • Corneal Confocal Microscopy: …
  - • Phenothiazine antioxidants in…
  - • Automated functional upper li…
- September (5)
- August (6)
  - • Test-retest reliability of an…
  - • Two funded post-doctoral posi…
  - • Nonataxia symptoms in Friedre…
  - • Activating frataxin expressio…
  - • Novel Nrf2-Inducer Prevents M…
  - • Longitudinal analysis of cont…
- July (7)
  - • RNA-Dependent Epigenetic Sile…
  - • Zinc(II) binding on human wil…
  - • PTC Therapeutics to acquire A…
  - • Young Investigators, Call for…
  - • Impact of Drosophila Models i…
  - • Autonomic function testing in…
  - • Structural characteristics of…
- June (5)
- May (5)
- April (3)
- March (9)
  - • Identification of p38 MAPK as…
  - • Iron regulatory protein defic…
  - • Mitofusin-Dependent ER Stress…
  - • GAA•TTC repeat expansion in…
  - • Pitfalls in molecular diagnos…
  - • Bone marrow transplantation s…
  - • Biophysical characterisation …
  - • Small RNA-seq analysis of cir…
  - • Progress in the treatment of …
- February (3)
- January (7)
  - • Activation of Frataxin Protei…
  - • Join Reata WebEx. Get updates…
  - • Quantitative proteomics in Fr…
  - • The role of Nrf2 signaling in…
  - • Effect Of Diazoxide on Friedr…
  - • Emotion Recognition and Psych…
  - • Friedreich and dominant ataxi…
2017 (79)
- December (8)
  - • Liquid Chromatography-High Re…
  - • Early VGLUT1-specific paralle…
  - • Somatic instability of the ex…
  - • Inducible and reversible phen…
  - • Mitochondrial pore opening an…
  - • The role of oxidative stress …
  - • Synthetic transcription elong…
  - • C-Path and FARA announce coll…
- November (5)
- October (16)
  - • Agilis Biotherapeutics Announ…
  - • Can rehabilitation improve th…
  - • Transplantation of wild-type …
  - • Friedreich's ataxia: clinical…
  - • Nrf2-Inducers Counteract Neur…
  - • Reata Announces First Patient…
  - • Friedreich Ataxia: Developmen…
  - • Sustained FXN expression in d…
  - • BioMarin Highlights Breadth o…
  - • CRISPR Therapeutics Awarded G…
  - • IARC 2017 Follow Up
  - • The Subclinical Cardiomyopath…
  - • FDA awards six grants for nat…
  - • Frataxin-deficient neurons an…
  - • Urinary, bowel and sexual sym…
  - • Agilis Biotherapeutics Update…
- September (6)
  - • Molecular Alterations in a Mo…
  - • Lipophilic methylene violet a…
  - • Reversible Axonal Dystrophy b…
  - • Impact of diabetes in the Fri…
  - • LNA effects on DNA binding an…
  - • Defining the Architecture of …
- August (10)
  - • Peptide SS-31 upregulates fra…
  - • Overexpression of Drosophila …
  - • Part 2 of the Phase II MOXIe …
  - • Cerebral Abnormalities in Fri…
  - • Jupiter Orphan Therapeutics R…
  - • Selected missense mutations i…
  - • FDA Confirms That Use of mFAR…
  - • Heart and Nervous System Path…
  - • Mitochondrial dysfunction in …
  - • Chondrial Announces FDA Orpha…
- July (6)
  - • Pharmacological therapeutics …
  - • Nicotinamide mononucleotide r…
  - • Call for Abstracts - 10th Ann…
  - • AFAF and FARA invite proposal…
  - • Circulating miR-323-3p is a b…
  - • How does performance of the F…
- June (2)
  - • Friedreich's ataxia induced p…
  - • Reata Pharmaceuticals, Inc. A…
- May (8)
  - • Cerebral compensation during …
  - • Progressive mitochondrial pro…
  - • Structural signature of class…
  - • Erythropoietin and small mole…
  - • Dimethyl Fumarate Mediates Nr…
  - • Normalization of timed neurop…
  - • Dysphagia in Friedreich Ataxi…
  - • Mesenchymal Stem Cell-Derived…
- April (3)
- March (4)
- February (4)
- January (7)
  - • Cardiac transplantation in Fr…
  - • Newly Funded FARA Grants
  - • Friedreich Ataxia: Hypoplasia…
  - • STEADFAST Background for the …
  - • Cytokine therapy-mediated neu…
  - • Deletion of the GAA repeats f…
  - • Mitochondrial capacity, muscl…
2016 (87)
- December (3)
- November (4)
- September (9)
  - • A longitudinal study of the S…
  - • Progression of Friedreich ata…
  - • Retrotope announces Phase I/I…
  - • Alleviating GAA repeat induce…
  - • Cognition in Friedreich's ata…
  - • Friedreich ataxia induced plu…
  - • Functional and Gait Assessmen…
  - • Pharmacological treatments fo…
  - • Neurobehavioral deficits in t…
- August (9)
  - • Serum versus Imaging Biomarke…
  - • Lentivirus-meditated frataxin…
  - • Frataxin silencing alters mic…
  - • Architecture of the Human Mit…
  - • Cerebral and cerebellar grey …
  - • Voice in Friedreich Ataxia
  - • Long-term treatment with thia…
  - • Agilis Biotherapeutics Announ…
  - • Pfizer aims to become industr…
- July (9)
  - • 9th Annual Friedreich’s Ata…
  - • The Replication of Frataxin G…
  - • Metal Homeostasis Regulators …
  - • The significance of intercala…
  - • Interferon gamma may improve …
  - • Deep sequencing of mitochondr…
  - • Crystal Structure of Bacillus…
  - • Downregulation of GSTK1 Is a …
  - • Vestibulo-ocular reflex dynam…
- June (3)
- May (11)
  - • Mitochondrial energy imbalanc…
  - • Reata Pharmaceuticals, Inc. A…
  - • Purkinje cell injury, structu…
  - • Plasma circulating cell-free …
  - • Horizon Pharma plc to Acquire…
  - • Abundance and Significance of…
  - • Meeting Notice: FARA's Bioma…
  - • Job Opportunity: Postdoctoral…
  - • Dorsal root ganglia in Friedr…
  - • Horizon Pharma plc Completes …
  - • Frataxin and the molecular me…
- April (11)
  - • Time-resolved functional anal…
  - • Two different pathogenic mech…
  - • Development of an AAV9 coding…
  - • Comorbid Medical Conditions i…
  - • Lymphoblast Oxidative Stress …
  - • Effects of Genetic Severity o…
  - • Using human pluripotent stem …
  - • Gastrocnemius and soleus spas…
  - • The Structure of the Complex …
  - • Clinical Experience With Defe…
  - • Iron chelation in the treatme…
- March (4)
- February (8)
  - • Reversal of epigenetic promot…
  - • Combined Cerebellar Proton MR…
  - • Intrathecal delivery of frata…
  - • Long-term effect of epoetin a…
  - • Retrotope advances RT001 in c…
  - • Gene transfer of brain derive…
  - • Activating frataxin expressio…
  - • Avalanche Biotechnologies and…
- January (16)
  - • Emerging therapies in Friedre…
  - • Friedreich Ataxia and nephrot…
  - • Nonneurological Involvement i…
  - • Pathology of Intercalated Dis…
  - • FARA Announces Catabasis Phar…
  - • Update on the STEADFAST study
  - • Chondrial Therapeutics Announ…
  - • Jupiter Orphan Therapeutics, …
  - • FARA Grant Program Update
  - • Annapurna Therapeutics to Col…
  - • Compound heterozygous FXN mut…
  - • Longitudinal magnetic resonan…
  - • Perturbation of cellular prot…
  - • Analyzing the Effects of a G1…
  - • GIFT-1, a phase IIa clinical …
  - • Efficient attenuation of Frie…
2015 (72)
- December (2)
  - • Retrotope RT0001 Clinical Tri…
  - • Reata RTA 408 Clinical Trial …
- November (2)
  - • Retrotope announces opening o…
  - • Funding Announcement: Clinica…
- September (8)
  - • A 22-Year Follow-up Study of …
  - • Expanded GAA repeats impede t…
  - • FXN Promoter Silencing in the…
  - • Delayed-onset Friedreich's at…
  - • Evidence for chromosome fragi…
  - • Frataxin levels in peripheral…
  - • Friedreich ataxia in Norway -…
  - • Investigation of mitochondria…
- August (12)
  - • Stable isotopes and LC-MS for…
  - • Phenotypic Screening for Frie…
  - • Key Patent Granted For AAVLif…
  - • New funding opportunity annou…
  - • Call for Abstracts due Friday…
  - • Targeting lipid peroxidation …
  - • Mitigation of Myocardial Isch…
  - • The Role of Frataxin in Doxor…
  - • Regional Cerebral Disease Pro…
  - • Gene Expression Profile in Pe…
  - • TORC1 Inhibition by Rapamycin…
  - • Ultra-structural hair alterat…
- July (3)
- June (11)
  - • New cellular model to follow …
  - • Novel aberrant genetic and ep…
  - • Src inhibitors modulate frata…
  - • Cardiomyopathy in FA: New bio…
  - • Turning E. coli into a Fratax…
  - • Turning Yeast into a Frataxin…
  - • Switching Frataxin Function b…
  - • Sensorimotor skills in Mouse …
  - • Iron-sulfur Assembly Complex …
  - • Interaction of frataxin with …
  - • Delivery of human frataxin ge…
- April (10)
  - • An open-label trial in Friedr…
  - • Friedreich's ataxia - a case …
  - • Expanded GAA repeats impair F…
  - • Excision of expanded GAA repe…
  - • Mitoferrin modulates iron tox…
  - • A longitudinal study of the F…
  - • Diffusion tensor imaging and …
  - • Structural characterization o…
  - • Mechanism of Action of 2-Amin…
  - • Publication for open-label tr…
- March (7)
  - • Iron loading site on the Fe-S…
  - • Abnormal Cochlear Potentials …
  - • Selective observation of the …
  - • Sensorimotor skills in Fxn KO…
  - • Excision of expanded GAA repe…
  - • The pathogenesis of cardiomyo…
  - • GSK Discovery Fast Track Chal…
- February (11)
  - • Ferredoxin, in conjunction wi…
  - • A novel GAA repeat expansion-…
  - • Milestones in Friedreich atax…
  - • Very late-onset friedreich at…
  - • Applying the Retro-Enantio Ap…
  - • Iron regulatory protein 1 sus…
  - • International Congress on "Fr…
  - • Frataxin inactivation leads t…
  - • Genome-Engineering Tools to E…
  - • Mammalian frataxin directly e…
  - • "Both Sides of the Wheelchair…
- January (6)
  - • Friedreich Ataxia: Failure of…
  - • Gait and Balance in Adults wi…
  - • Biological and clinical chara…
  - • Friedreich's ataxia: the Euro…
  - • Unveiling a common mechanism …
  - • Frataxin knockdown in human a…
2014 (70)
- December (5)
- November (3)
- October (7)
  - • Open-label pilot study of int…
  - • An AAV9 coding for frataxin c…
  - • Probing the Kinetic Stabiliti…
  - • Comparing speech characterist…
  - • Nasality in Friedreich ataxia
  - • Overlapping Binding Sites of …
  - • A longitudinal VBM study moni…
- September (10)
  - • Iron-sulfur cluster biogenesi…
  - • Understanding the frustration…
  - • Cellular, Molecular and Funct…
  - • Overlapping Binding Sites of …
  - • Frataxin activates Fe-S clust…
  - • A 7-year-old girl with hypert…
  - • Epigenetic therapy for Friedr…
  - • Deferiprone in Friedreich ata…
  - • Epigenetic promoter silencing…
  - • A study of up to 12 years o…
- August (11)
  - • Dyclonine rescues frataxin de…
  - • Frataxin deficiency increases…
  - • Post-Doctoral Fellowship avai…
  - • Mesenchymal Stem Cells Improv…
  - • Human Frataxin Activates Fe-S…
  - • Therapeutic approaches for th…
  - • Sensitivity of Spatiotemporal…
  - • Functional Characterization o…
  - • The role of frataxin in fissi…
  - • MutLα Heterodimers Modify th…
  - • Myelin paucity of the superio…
- June (5)
- May (10)
  - • Cross-sectional analysis of g…
  - • Atypical Friedreich ataxia in…
  - • Nicotinamide in Friedreich's …
  - • Epigenetic and neurological e…
  - • R-loops Associated with Tripl…
  - • Dentate nuclei T2 relaxometry…
  - • Pathophysiogical and therapeu…
  - • Cell and gene therapy for Fri…
  - • Saccade reprogramming in Frie…
  - • Frataxin deficiency in neonat…
- April (5)
- March (4)
- February (4)
- January (6)
  - • Usefulness of frataxin immuno…
  - • Beyond loss of frataxin: the …
  - • Frataxin-bypassing Isu1: char…
  - • The kinetics of folding of fr…
  - • HFE p.C282Y heterozygosity is…
  - • Dysphagia and swallowing-rela…
2013 (9)
- December (4)
- October (3)
- September (2)
  - • EFACTS - European Friedreich'…
  - • FARA, Ataxia UK and GoFAR inv…

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