Antibiotics that enable ribosomes to "read through" premature stop codons (nonsense mutations), which truncate proteins, may kick-start a new approach to gene therapy. A team of researchers from the University of Alabama at Birmingham and from the Women's and Children's Hospital in South Australia applied a long-ago observation about the antibiotic gentamicin on ribosomes to correct the enzyme deficiency that causes Hurler syndrome, in cultured fibroblasts from patients.1 The antibiotic apparently enables ribosomes to zip past nonsense mutations, in which a single base substitution converts an amino-acid encoding codon into a stop codon, shortening the protein product. "It's been known since the early 1960s that aminoglycoside antibiotics bind the 16S bacterial ribosomal RNA subunit, affecting translational fidelity, causing read-through of stop codons in bacteria. In mammalian ribosomes, the region is similar but it has a lower binding affinity, and that's why these antibiotics kill bacteria before they kill people....
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