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Trends in Pharmacological Sciences|March 26, 2019
Therapeutic Prospects for Friedreich's AtaxiaSiyuan Zhang, Marek Napierala, Jill S Napierala
Stem Cell Research & Therapy|January 9, 2026
Human pluripotent stem cell models of Friedreich's ataxia: innovations, considerations, and future perspectivesHa Thi Nguyen, Marek Napierala, Jill S Napierala
Stem Cell Research|June 27, 2024
Generation of genetically modified Friedreich's ataxia induced pluripotent stem cell lines and isogenic control lines carrying an inducible neurogenin-2 expression cassetteSara Miellet, Marnie Maddock, Jill S Napierala, et al.
Expert Opinion on Orphan Drugs|March 5, 2019
Progress in understanding Friedreich's ataxia using human induced pluripotent stem cellsAnna M Schreiber, Julia O Misiorek, Jill S Napierala, et al.
The CRISPR Journal|November 4, 2020
A CRISPR-Cas9, Cre-lox, and Flp-FRT Cascade Strategy for the Precise and Efficient Integration of Exogenous DNA into Cellular GenomesJixue Li, Yanjie Li, Kevin M Pawlik, et al.
Stem Cell Research|August 26, 2019
Excision of the expanded GAA repeats corrects cardiomyopathy phenotypes of iPSC-derived Friedreich's ataxia cardiomyocytesJixue Li, Natalia Rozwadowska, Amanda Clark, et al.
Frontiers in Neuroscience|March 14, 2022
Selected Histone Deacetylase Inhibitors Reverse the Frataxin Transcriptional Defect in a Novel Friedreich's Ataxia Induced Pluripotent Stem Cell-Derived Neuronal Reporter SystemAnna M Schreiber, Yanjie Li, Yi-Hsien Chen, et al.
Human Gene Therapy|June 13, 2020
Defining Transcription Regulatory Elements in the Human Frataxin Gene: Implications for Gene TherapyJixue Li, Yanjie Li, Jun Wang, et al.
Plos One|December 21, 2017
Somatic instability of the expanded GAA repeats in Friedreich's ataxiaAshlee Long, Jill S Napierala, Urszula Polak, et al.
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.
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