CRISPR genetic manipulation has a similar parallel in which molecular scissors are used, and selected portions of DNA are cut. This comparison is a favorite of Stanley Qi, bioengineering assistant professor at Stanford.
“CRISPR can be as simple as a cutter or more advanced as a regulator, an editor, a labeler, or imager. Many applications are emerging from this exciting field,” explained assistant professor Qi. Nevertheless, many other CRISPR technologies that are currently used or clinically studied for the brain, eye, or liver genetic therapy stay restricted since they all have the same defect: overly big and thus too difficult to transport to cells.
Qi and his colleagues reveal something they consider to be a big stride forward for CRISPR in a study issued in Molecular Cell on Friday. They found an effective, multifunctional mini-CRISPR mechanism. The typical CRISPR systems, known as Cas9 and Cas12a, are composed of roughly 1000 to 1,500 amino acids and have 529 CRISPR-related proteins (Cas).
In tests, the scientists found that CasMINI, similarly to its heavier siblings, was able to remove, trigger and modify genetic coding. Its reduced size makes it simpler to transport to people or human cells. Therefore, it can be considered a possible instrument to cure many illnesses such as eye disorders, degenerate organs, and general hereditary diseases.
“This ability to engineer these systems has been desired in the field since the early days of CRISPR, and I feel like we did our part to move toward that reality. And this engineering approach can be so broadly helpful. That is what excites me—opening the door on new possibilities,” said Qi
The scientists started working with some other researchers on genetic therapy. The team is also focused on contributing to the progress of RNA technology, similar to the development of the mRNA vaccine for Coronavirus.