Multipurpose Mini CRISPR System Easier to Deliver Into Human Cells for Gene Therapy, Treat Rheumatoid Arthritis, Diverse Ailments

Bioengineers reimagine the CRISPR system as a Swiss Army knife packed with many applications. They repurposed it to make a mini version, called CasMINI, that can easily be delivered into human cells for gene therapy and treat various ailments.

The new technology, described in the study titled "Engineered Miniature CRISPR-Cas System for Mammalian Genome Regulation and Editing", published in the journal Molecular Cell, shows a more effective and efficient way of cell engineering.

The Powerful Gene Editing Tool CRISPR System

Stanley Qi, the lead author of the study and an assistant professor of bioengineering at Stanford University, said that CRISPR gene-editing system was always associated with molecular scissors that cut out some sections of DNA. But for him, it is a Swiss Army knife wherein various applications have arisen from this exciting field.

According to EurekAlert! the development of CRISPR-Cas systems for human cells has revolutionized genome engineering as it gave opportunities to develop gene therapies for various genetic diseases. CRISPR-Cas systems have been clinically tested for gene therapy in the eye, brain, and liver.

But their scope remains limited because they are too large and too hard to deliver into human cells of living organisms. As a result, there is a need to develop smaller, highly efficient, and effective Car systems to facilitate next-generation cell engineering and gene therapy applications.

CasMINI: The Smallest CRISPR System to Date

Bioengineers from Stanford University developed a smaller CRISPR-Cas system called CasMINI to address size in CRISPR-Cas9 and its derivatives, UPI reported.

Qi said that this new technology is a critical step forward in enhancing CRISPR system genome-engineering applications. To date, it is the smallest CRISPR system that works like a Swiss Army knife rather than molecular scissors, such as CRISPR-Cas9. Qi added that their research presents a miniature version that is highly portable for easy use.

The tea pointed out this new technology will allow other scientists to develop novel gene therapies that otherwise would not be possible using previous CRISPR systems because of their larger size.

Shrinking Existing CRISPR System

According to SciTech Daily, researchers started with the CRISPR protei Cas12f, also known as Cas14, to make the system shrink. Cas 14 contains only about 400 to 700 amino acids, but it is not well-suited to mammalian cells. Nevertheless, scientists find it exciting to experiment with.

For the first year, they did not saw any activity of the natural Cas12f in human cells. But alterations through bioengineering saw some engineered proteins start to improve in performance, pushing the team to move forward to engineer the RNA that guides the Cas to the DNA of the targeted cells.

Then they decided to stop at some moment as they have already made a good system out of a nonworking CRISPR. Now, they have to think of applications for this mini CRISPR system to work in human cells.

They tested its ability to delete and edit genes in human cells in the lab, including genes related to anti-tumor response, anemia, and HIV infection. The team found that it worked on almost every gene they tested, with robust responses in most of them. They are now working with other scientists to pursue gene therapies for other ailments and create mRNA-based COVID-19 vaccines.

Check out more news and information on CRISPR in Science Times.