Just this week there have been reports of using the CRISPR gene-editing tool to alter a number of disease-linked genes. A report in the Proceedings of the National Academy of Sciences on Monday described the tool's use to disable a glaucoma mutation, while another in Protein & Cell targeted a β-thalassemia mutation.
New Scientist writes that a whole host of diseases could be treated using CRISPR, particularly if cells could be edited while still in patients' bodies. The stumbling block there is getting the bulky CRISPR machinery into cells, it says. But, it notes that companies like Intellia Therapeutics have been working on ways to introduce the CRISPR molecules using fatty molecules into the liver, while the University of California, Berkeley, researchers have turned to delivery via gold nanoparticles and researchers like at University of Washington have been using adeno-associated virus delivery.
The various delivery approaches have their drawbacks, New Scientist says. If the CRISPR cutting proteins are delivered to cells, such as by gold nanoparticle, they break down quickly, but if the DNA encoding the proteins is instead delivered, as in the adeno-associated virus approach, the proteins continue to be made, increasing the chances of off-target effects. But, there, too, it adds that researchers are working on giving the CRISPR protein a shelf life so they don't hang about in the cell.