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Molecular Therapy. Nucleic Acids|July 24, 2025
Antisense oligonucleotide therapy for patients with Friedreich's ataxia carrying the c.165+5G>C splicing mutationPouiré Yameogo, Selina Aguilar, Thazha P Prakash, et al.
Neurobiology of Disease|January 13, 2023
Neurobehavioral deficits of mice expressing a low level of G127V mutant frataxinDaniel Fil, Robbie L Conley, Aamir R Zuberi, et al.
Cell Reports|July 19, 2016
Stalled DNA Replication Forks at the Endogenous GAA Repeats Drive Repeat Expansion in Friedreich's Ataxia CellsJeannine Gerhardt, Angela D Bhalla, Jill Sergesketter Butler, et al.
Disease Models & Mechanisms|September 11, 2023
Comparative multi-omic analyses of cardiac mitochondrial stress in three mouse models of frataxin deficiencyNicole M Sayles, Jill S Napierala, Josef Anrather, et al.
RNA (New York, N.Y.)|June 2, 2019
Efficient electroporation of neuronal cells using synthetic oligonucleotides: identifying duplex RNA and antisense oligonucleotide activators of human frataxin expressionXiulong Shen, Sharon Beasley, Jennifer N Putman, et al.
Human Molecular Genetics|June 16, 2022
Premature transcription termination at the expanded GAA repeats and aberrant alternative polyadenylation contributes to the Frataxin transcriptional deficit in Friedreich's ataxiaYanjie Li, Jixue Li, Jun Wang, et al.
Human Mutation|January 18, 2007
CAG and CTG repeat polymorphism in exons of human genes shows distinct features at the expandable lociMatylda Rozanska, Krzysztof Sobczak, Anna Jasinska, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy|March 12, 2015
Excision of Expanded GAA Repeats Alleviates the Molecular Phenotype of Friedreich's AtaxiaYanjie Li, Urszula Polak, Angela D Bhalla, et al.
Cell Stem Cell|November 3, 2010
Friedreich's ataxia induced pluripotent stem cells model intergenerational GAA⋅TTC triplet repeat instabilitySherman Ku, Elisabetta Soragni, Erica Campau, et al.
Future Science OA|July 10, 2019
The current state of biomarker research for Friedreich's ataxia: a report from the 2018 FARA biomarker meetingIan A Blair, Jennifer Farmer, Steven Hersch, et al.
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