In The News — 17 February 2014

CRISPR has allowed scientists to execute microsurgeries on not just bacteria genomes but also animal genomes. The CRISPR technology opens the gateway to identifying and curing genome defects and diseases. It has the potential to reverse the effects of diseases like sickle cell anemia, cystic fibrosis, and HIV.

This technology could allow researchers to perform microsurgery on genes, precisely and easily changing a DNA sequence at exact locations on a chromosome. Along with a technique called TALENs, invented several years ago, and a slightly older predecessor based on molecules called zinc finger nucleases, CRISPR could make gene therapies more broadly applicable, providing remedies for simple genetic disorders like sickle-cell anemia and eventually even leading to cures for more complex diseases involving multiple genes. Most conventional gene therapies crudely place new genetic material at a random location in the cell and can only add a gene. In contrast, CRISPR and the other new tools also give scientists a precise way to delete and edit specific bits of DNA—even by changing a single base pair. This means they can rewrite the human genome at will.

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Elan Meltzer

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