Učitavanje...
CRISPR-Cas9 Gene Editing of Hematopoietic Stem Cells from Patients with Friedreich’s Ataxia
Friedreich’s ataxia (FRDA) is an autosomal recessive neurodegenerative disorder caused by expansion of GAA repeats in intron 1 of the frataxin (FXN) gene, leading to significant decreased expression of frataxin, a mitochondrial iron-binding protein. We previously reported that syngeneic hematopoieti...
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| Izdano u: | Mol Ther Methods Clin Dev |
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| Glavni autori: | , , , , , , , |
| Format: | Artigo |
| Jezik: | Inglês |
| Izdano: |
American Society of Gene & Cell Therapy
2020
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| Teme: | |
| Online pristup: | https://ncbi.nlm.nih.gov/pmc/articles/PMC7240056/ https://ncbi.nlm.nih.gov/pubmed/32462051 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.omtm.2020.04.018 |
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