PLX085533

GSE99400: RNA-seq of GM15850 and GM15851 cells treated with synthetic transcription elongation factors.

• Organsim human
• Type RNASEQ
• Target gene
• Project ARCHS4

Switching a paused RNA polymerase II into productive elongation is tightly-regulated, especially at genes involved in human development and disease. To exert control on this rate-limiting step, we designed sequence-specific synthetic transcription elongation factors (Syn-TEFs). These molecules are composed of programmable DNA-binding ligands flexibly tethered to a small molecule that binds a component of the transcription elongation machinery. The resultant bifunctional molecules convert constituent modules from broad-spectrum inhibitors of transcription into a gene-specific stimulator of transcriptional elongation. Here, we present Syn-TEF1, a molecule that actively facilitates transcription across repressive GAA repeats that silence frataxin expression in Friedreichs ataxia, a debilitating and ultimately lethal neurodegenerative disease with no effective therapy. SOURCE: Aseem AnsariAnsari University of Wisconsin-Madison