In Nature this week, Broad Institute scientists describe an improved version of the genome-editing technology CRISPR/Cas9. Though widely used, the reliance of the approach on the relatively large Cas9 enzyme, which cuts DNA in specific locations, has restricted its use to cell lines and embryonic manipulation. The researchers were able to alter the system to use a smaller version of the Cas9 enzyme that was discovered in the bacteria Staphylococcus aureus. They were then able to use this new system to target a cholesterol regulatory gene in mice. GenomeWeb has more on this study here.
And in Nature Genetics, a team of French scientists reports the results of an exome sequencing analysis of 243 liver tumors that identified new mutational signatures and potential therapeutic targets for the hepatocellular carcinoma. They found mutational signatures associated with specific risk factors such as combined alcohol and tobacco consumption, as well as 161 putative driver genes associated with 11 recurrently altered pathways. In nearly 30 percent of tumors, the researchers also found genetic alterations potentially druggable by FDA-approved therapeutics. GenomeWeb also covers this study here.