For the first time, a synthetic compound has been made that can bind to DNA in the cells’ energy powerhouses, suppressing a gene associated with nerve and muscle disease. Pyrrole-imidazole polyamides (PIPs) are compounds that can read specific DNA sequences inside living cells and silence disease-causing genes. They prevent transcription factors, from binding to specific parts of the DNA strand, thus suppressing the transcription of DNA into RNA.
Most DNA is found in the nucleus. But mitochondria, the cell’s powerhouses, also host a small amount of DNA. PIPs can cross the nuclear membrane to bind to nuclear DNA, but are incapable of crossing the mitochondrial membrane...
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