Dubbed the Swiss Army Knife, this new technology is a development in technology known as CRISPR, first developed in 2012 and described as one of the most powerful medical instruments in history.
The technology works by eliminating specific sections of DNA. Experts agree that there is huge potential in CRISPR as an effective treatment, and the CRISPR gene-editing system is usually associated with Cas9 (an enzyme derived from bacteria that cuts and replaces faulty genes), but there is a problem that its efficiency is somewhat restricted by its relatively large size, making it difficult for the mechanism to squeeze cells and start working.
Scientists at the University of Stamford have now developed a CRISPR tool called CasMINI, using a protein close to Cas9, but much smaller than it, supposed to be able to enter human cells more easily.
The team of researchers made a very small enzyme called Cas14, which is half the size of Cas9.
But Cas14 doesn’t work in mammals, so Stamford’s team genetically manipulated the enzyme to fix this problem and called it CasMINI.
Dr. Lee (Stanley) Key, from the Stamford research team who worked on the project, said: “There have been previous efforts to improve CRISPR’s performance. This is a crucial step forward for CRISPR genome engineering applications. The work presents the youngest CRISPR to date, according to our knowledge, as a genome editing technique.”
He added: “If people sometimes think of Cas9 as molecular scissors, here we’ve made a Swiss knife with multiple functions. It’s not big, but it’s a portable miniature for ease of use.”
In their experiments, the researchers pointed out that CasMINI can delete, activate and modify the genetic code just like its larger counterparts. Its smaller size means it should be easier to deliver to human cells and the human body, making it a potential tool for treating diverse diseases, including eye diseases, organ regeneration, cancer treatment, and genetic diseases in general.
CRISPR technology is known as a serious game-changer in the medical field. That’s why Professors Emmanuel Charpentier and Jennifer Doudna, who co-discovered Cas9, won the Nobel Prize in Chemistry last year for their work in the field.
But so far, CRISPR use in humans has been very limited by a small set of studies.
Some people are concerned about the potential long-term effects of this type of technology, with safety and ethical implications being a particular concern.
There have been some examples of illegal use of CRISPR, such as disgraceful scientist Jiankoy, who used CRISPR on twin embryos of girls in an attempt to make them resistant to HIV.
But scientists are still unable to use this technology to combat some of the world’s most problematic diseases.
The study was published in detail in the journal Molecular Cell.