A team of researchers from the University of California, Berkeley, and the University of Tokyo have developed a new delivery system for CRISPR gene editing technology using lipid nanoparticles. The technology allows for more efficient and targeted delivery of CRISPR components to specific cells in the body.

CRISPR technology has the potential to revolutionize medicine by allowing researchers to edit the genetic code of cells, potentially curing genetic diseases. However, one of the major challenges of CRISPR is delivering the necessary components, including the Cas9 enzyme and the guide RNA, to the target cells without causing adverse effects.

The researchers developed a lipid nanoparticle delivery system that can efficiently encapsulate and protect the CRISPR components, while also allowing for targeted delivery to specific cells. The system is based on a new type of lipid nanoparticle called a charged multilamellar vesicle (cMLV), which is made up of multiple layers of lipids with a positive charge on the outer layer.

According to the lead author of the study, Kiana Aran, the cMLV delivery system is more effective than previous methods of CRISPR delivery. “Our cMLV system is unique in that it can efficiently encapsulate the Cas9 protein and guide RNA, protect them from degradation, and effectively deliver them to the target cells,” she said.

The researchers tested the cMLV delivery system in human embryonic kidney cells and found that it was able to deliver the CRISPR components to the cells with high efficiency and specificity. The system was also able to edit the genes of the target cells without causing any significant toxicity.

The cMLV delivery system has the potential to be used in a wide range of applications, including the development of new gene therapies for genetic diseases. The researchers are now working to optimize the technology for use in animal models and ultimately in clinical trials.

Overall, the development of a more efficient and targeted CRISPR delivery system is a significant step forward in the development of CRISPR-based therapies. The cMLV technology has the potential to unlock the full potential of CRISPR by allowing researchers to more effectively edit the genes of specific cells in the body, potentially curing genetic diseases and revolutionizing the field of medicine.

Via The Impactlab