Here are four things to know about the technological development.
1. CRISPR, which stands for Clustered Regularly Interspaced Short Palindromic Repeats, is a gene-editing technology that enables scientists to edit an organism’s DNA. The CRISPR-Cas9 system — which creates modified RNA segments that bind to the CRISPR-associated protein 9 enzyme — allows scientists to add, remove and replace whole genes.
2. In two separate studies published Oct. 25, researchers from the Broad Institute discussed their work with “base editing,” a modified version of CRISPR used to edit DNA and RNA. With base editing, scientists can modify smaller segments of a person’s genome, such as point mutations, in which only one base pair in a strand of DNA necessitates intervention.
3. One of the studies, published in Nature, used base editing to rearrange the atoms in a chemical base A to resemble a chemical base G. The study was led by David Liu, PhD, a chemistry professor at Cambridge, Mass.-based Harvard University and member of the Broad Institute. His team has used the technique to produce a mutation that restrains sickle-cell anemia in humans.
4. The base editing technique has the potential to intervene in a range of genetic disorders. Point mutations comprise 32,000 of the 50,000 human genome changes that are associated with diseases, according to the MIT Technology Review. Dr. Liu’s lab will continue to investigate the potential of base editing to address conditions like blood disorders, neurological disorders and hereditary deafness.
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