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CRISPR-Cas9 Gene Editing of Hematopoietic Stem Cells from Patients with Friedreich's Ataxia: Molecular Therapy - Methods & Clinical Development
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Advantages and Limitations of Gene Therapy and Gene Editing for Friedreich's Ataxia | Semantic Scholar
![FA Research Alliance on Twitter: "We all have the FXN gene, but those with FA have a specific DNA mutation that causes FA. The most common is GAA. http://t.co/RBNECkNGPo" / Twitter FA Research Alliance on Twitter: "We all have the FXN gene, but those with FA have a specific DNA mutation that causes FA. The most common is GAA. http://t.co/RBNECkNGPo" / Twitter](https://pbs.twimg.com/media/Bw3YnEJIcAE5xTy.jpg)
FA Research Alliance on Twitter: "We all have the FXN gene, but those with FA have a specific DNA mutation that causes FA. The most common is GAA. http://t.co/RBNECkNGPo" / Twitter
![High Levels of Frataxin Overexpression Lead to Mitochondrial and Cardiac Toxicity in Mouse Models: Molecular Therapy - Methods & Clinical Development High Levels of Frataxin Overexpression Lead to Mitochondrial and Cardiac Toxicity in Mouse Models: Molecular Therapy - Methods & Clinical Development](https://www.cell.com/cms/asset/ad5e32bf-6122-471c-921e-94e6e9b686c8/fx1.jpg)
High Levels of Frataxin Overexpression Lead to Mitochondrial and Cardiac Toxicity in Mouse Models: Molecular Therapy - Methods & Clinical Development
![Gene regulation and epigenetics in Friedreich's ataxia - Yandim - 2013 - Journal of Neurochemistry - Wiley Online Library Gene regulation and epigenetics in Friedreich's ataxia - Yandim - 2013 - Journal of Neurochemistry - Wiley Online Library](https://onlinelibrary.wiley.com/cms/asset/40192b0c-d0cf-40fa-bb4c-f4725cfe24ba/jnc12254-fig-0001-m.jpg)
Gene regulation and epigenetics in Friedreich's ataxia - Yandim - 2013 - Journal of Neurochemistry - Wiley Online Library
![Structure of the FXN gene and transcription maps. The structures of the... | Download Scientific Diagram Structure of the FXN gene and transcription maps. The structures of the... | Download Scientific Diagram](https://www.researchgate.net/publication/41434552/figure/fig11/AS:668543804788747@1536404672777/Structure-of-the-FXN-gene-and-transcription-maps-The-structures-of-the-FXN-gene-except.png)
Structure of the FXN gene and transcription maps. The structures of the... | Download Scientific Diagram
![Beyond Loss of Frataxin: the Complex Molecular Pathology of Friedreich Ataxia - Marguerite V Evans-Galea - Discovery Medicine Beyond Loss of Frataxin: the Complex Molecular Pathology of Friedreich Ataxia - Marguerite V Evans-Galea - Discovery Medicine](https://www.discoverymedicine.com/Marguerite-V-Evans-Galea/files/2014/01/discovery_medicine_no_91_marguerite_v_evans_galea_figure_1.jpg)
Beyond Loss of Frataxin: the Complex Molecular Pathology of Friedreich Ataxia - Marguerite V Evans-Galea - Discovery Medicine
![Schematic diagram of the FXN and GFP genes containing intronic GAA·TTC... | Download Scientific Diagram Schematic diagram of the FXN and GFP genes containing intronic GAA·TTC... | Download Scientific Diagram](https://www.researchgate.net/publication/23285945/figure/fig8/AS:324926885122065@1454480014597/Schematic-diagram-of-the-FXN-and-GFP-genes-containing-intronic-GAATTC-repeats-A.png)
Schematic diagram of the FXN and GFP genes containing intronic GAA·TTC... | Download Scientific Diagram
![Needles&Neurons on Twitter: "In the FXN gene, a GAA sequence repeats between 7-22 times. In Friedreich ataxia, this GAA triplet of DNA code is expanded, sometime repeated hundreds of times due to Needles&Neurons on Twitter: "In the FXN gene, a GAA sequence repeats between 7-22 times. In Friedreich ataxia, this GAA triplet of DNA code is expanded, sometime repeated hundreds of times due to](https://pbs.twimg.com/media/E_pegcZXIAURfho.jpg)
Needles&Neurons on Twitter: "In the FXN gene, a GAA sequence repeats between 7-22 times. In Friedreich ataxia, this GAA triplet of DNA code is expanded, sometime repeated hundreds of times due to
![IJMS | Free Full-Text | The NRF2 Signaling Network Defines Clinical Biomarkers and Therapeutic Opportunity in Friedreich's Ataxia IJMS | Free Full-Text | The NRF2 Signaling Network Defines Clinical Biomarkers and Therapeutic Opportunity in Friedreich's Ataxia](https://www.mdpi.com/ijms/ijms-21-00916/article_deploy/html/images/ijms-21-00916-g001.png)
IJMS | Free Full-Text | The NRF2 Signaling Network Defines Clinical Biomarkers and Therapeutic Opportunity in Friedreich's Ataxia
Structure–Function Analysis of Friedreich's Ataxia Mutants Reveals Determinants of Frataxin Binding and Activation of the Fe–S Assembly Complex | Biochemistry
![The FXN gene location on chromosome 9 at position 21.11 (from reference [4 | Download Scientific Diagram The FXN gene location on chromosome 9 at position 21.11 (from reference [4 | Download Scientific Diagram](https://www.researchgate.net/publication/319560725/figure/fig1/AS:730880179961859@1551266822671/The-FXN-gene-location-on-chromosome-9-at-position-2111-from-reference-4.png)