Day by day, the usage of the new gene editing device, that is, as CRISPR/Cas9 is increasing as it has shown potential for treating genetic disorders, including muscular dystrophy, cystic fibrosis, and hemophilia. This technique was isolated from bacteria. But foe its function, the device should be transported safely through the cell membrane and into the cell’s nucleus. This is usually an intricate process as there is a possibility of the CRISPR/Cas9 to be trapped by the defense mechanism of a cell and this reduces its treatment capability.
Recently, Vincent Rotello, an expert in nanochemistry, has developed a delivery system with the use of nanoparticles to help CRISPR/Cas9 travel through the membrane and then into the nucleus of the cell and at the same time, preventing entrapment by the cellular mechanism.
CRISPR has two elements. First, a scissor-like Cas9 protein, named Cas9En, and second, an RNA molecule that is known as sgRNA. The sgRNA directs the Cas9 to its destination gene. When the Cas9–sgRNA complex reaches the specific target gene in the cell’s nucleus, it can cross-examine the gene’s genetic errors and then repair them with the aid of the repair mechanism of the host cell.
Rotello said, “By fine-tuning the communications between nanoparticles and engineered Cas9En protein, it was possible for us to design these delivery vectors. When the vectors hauling the sgRNA and Cas9 protein make a contact with the cell membrane, it fuses and liberates the Cas9–sgRNA into the cytoplasm of the cell.”
Rotello further continued saying, “Cas9 protein also presents a nuclear guiding sequence that guides the pair into the targeted nucleus. This can be done by tweaking the Cas9 protein. We were successful in delivering the Cas9–sgRNA complex into the nucleus of the cell without being trapped during its travel. We have observed the delivery process in real time with the help of a sophisticated microscopy.”
The researchers are also hoping to use Cas9En as an interface for delivery of several other materials, including lipid nanoparticles, self-assembling peptides or polymers.
This new technique can surely open new avenues for treating various diseases. Feel free to share your views regarding the same.