ArticleDisease models & mechanisms2015
A new cellular model to follow Friedreich's ataxia development in a time-resolved way.
Article in Disease models & mechanisms, 2015. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 10 papers.
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Who cites it
10 citing papers in PubMed, 14 citations in OpenAlex.
- Label-Free, Real-Time Measurement of Metabolism of Adherent and Suspended Single Cells by In-Cell Fourier Transform Infrared Microspectroscopy.International journal of molecular sciences · 2021Article
- A new tool to determine the cellular metabolic landscape: nanotechnology to the study of Friedreich's ataxia.Scientific reports · 2019Article
- Next Generation Precision Medicine: CRISPR-mediated Genome Editing for the Treatment of Neurodegenerative Disorders.Journal of neuroimmune pharmacology : the official journal of the Society on NeuroImmune Pharmacology · 2019Review
- Adding a temporal dimension to the study of Friedreich's ataxia: the effect of frataxin overexpression in a human cell model.Disease models & mechanisms · 2018Article
- An efficient platform for generating somatic point mutations with germline transmission in the zebrafish by CRISPR/Cas9-mediated gene editing.The Journal of biological chemistry · 2018Article
- The role of oxidative stress in Friedreich's ataxia.FEBS letters · 2018Review
- The time dimension of neurodegeneration: the example of Friedreich's ataxia.Journal of neurology & neuromedicine · 2017Article
- Applications of CRISPR/Cas9 for Gene Editing in Hereditary Movement Disorders.Journal of movement disorders · 2016Review
- Time-resolved functional analysis of acute impairment of frataxin expression in an inducible cell model of Friedreich ataxia.Biology open · 2016Article
- Genome Engineering with TALE and CRISPR Systems in Neuroscience.Frontiers in genetics · 2016Review
Corrections and comments
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Authors and funding
6 authors at 2 institutions in 1 country.
Funding
Abstract
Friedreich's ataxia (FRDA) is a recessive autosomal ataxia caused by reduced levels of frataxin (FXN), an essential mitochondrial protein that is highly conserved from bacteria to primates. The exact role of frataxin and its primary function remain unclear although this information would be very valuable to design a therapeutic approach for FRDA. A main difficulty encountered so far has been that of establishing a clear temporal relationship between the different observations that could allow a distinction between causes and secondary effects, and provide a clear link between aging and disease development. To approach this problem, we developed a cellular model in which we can switch off/on in a time-controlled way the frataxin gene partially mimicking what happens in the disease. We exploited the TALEN and CRISPR methodologies to engineer a cell line where the presence of an exogenous, inducible FXN gene rescues the cells from the knockout of the two endogenous FXN genes. This system allows the possibility of testing the progression of disease and is a valuable tool for following the phenotype with different newly acquired markers.
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Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.