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Charles Plessy @charles_plessy
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An entirely factor was developped by Erwin, Ansari (+others) at the University of Wisconsin-Madison (+elsewhere).

doi.org/10.1126/science.aan641

It binds DNA through a pyrrole- and imidazole-based polyamide specific for the sequence AAGAAGAAG, which is dramatically expanded in the genome of Friedreich's ataxia's patients.

Its other half is a drug (JQ1) that recruits the transcription elongation factor BRD4, which rescues expression of the mutated gene.

Very impressive.

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