| Name | Syn-TEF1 |
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| Description | Syn-TEF1 is a molecule actively enables transcription across repressive GAA repeats that silence frataxin expression in Friedreich's ataxia, license transcription elongation at targeted genomic loci; targets the DNA sequence 5'-AAGAAGAAG-3', activate FXN expression in primary patient cells and patient-derived fibroblasts, iPSCs, cardiomyocytes, sensory neurons, and mouse xenografts. |
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| References | References 1. Erwin GS, et al. Science. 2017 Dec 22;358(6370):1617-1622. View Related Products by Target Other Targets |
| Molecular Formula | C77H104ClN23O16S |
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| Molecular Weight | 1675.334 |