Researchers have developed tiny nanocapsules that show promise as an alternative way to deliver gene-editing tools for treating disease.
Most gene editing is done with viral vectors, which are highly efficient at delivering genes to their target. But they can carry risks, including undesirable immune responses, and they are complicated to manufacture.
In their search for better a delivery method, researchers developed a nano-sized polymer shell—thousands of times smaller than the width of a human hair—that they said is capable of carrying a payload for gene editing. The surface of the nanocapsule can be equipped with special chemicals that allow it to target certain cell types. The researchers equipped the new nanocapsule, which is biodegradable, with a version of the gene-editing tool CRISPR-Cas9 and tested it in animal tissues. The results, they said, were encouraging. The study, which received some funding support from NHLBI, appears in Nature Nanotechnology.