Syn-TEF1

Names

[ Name ]:
Syn-TEF1

Biological Activity

[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.

[References]

References 1. Erwin GS, et al. Science. 2017 Dec 22;358(6370):1617-1622. View Related Products by Target Other Targets

Chemical & Physical Properties

[ Molecular Formula ]:
C₇₇H₁₀₄ClN₂₃O₁₆S

[ Molecular Weight ]:
1675.334

Related Compounds

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