A CRISPR gene boost reduces seizures and strengthens muscles in mice
Some genetic diseases leave cells without a crucial protein. One possible treatment is to persuade genes already inside those cells to work harder, but the necessary machinery can be difficult to deliver, especially when researchers want to target several genes at once. A new compact CRISPR system devised by Hainan Zhang of the Shanghai Institute of Materia Medica, Chinese Academy of Sciences,…
A new CRISPR activation system, developed by researchers at the Shanghai Institute of Materia Medica, allows multiple gene-targeting instructions to be delivered in a single viral package. This compact system, called smCRISPRa, has shown promising results in treating genetic diseases in mice. In experiments with a Dravet syndrome model, the treatment reduced seizures and prevented early deaths, while in a Duchenne muscular dystrophy model, it improved muscle function for six months.
The system uses a small CRISPR protein called Cas12m, which can also cut RNA guides into separate working pieces inside a cell, allowing several guides to share one compact set of instructions. The researchers created a platform that allows multiple guides to focus its activity on one gene or distribute it across several. While the study shows promising results, further testing is needed to evaluate safety and off-target effects before clinical translation.
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