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Repairing the Genome's Spelling Mistakes

GENETICSResearchers have recently devised a new method that may provide a better way of correcting the gene defects that cause hereditary human diseases such as sickle cell anemia. Working with both cultured cells and experimental animals, they've shown that they can remedy defects caused by single DNA spelling mistakes using short lengths of synthetic DNA and RNA called chimeras. These molecules are designed to contain DNA with the correct version of the misspelled letter flanked by RNA that perfectly mirrors the rest of the target gene segment. By pairing up with the defective gene, the chimeras can trick the cell's DNA repair machinery into replacing the wrong nucleotide in a gene with the right one. If the technique, which is called chimeraplasty, works in human patients, it could offer some significant advantages over current gene therapy strategies.