CRISPR Technology May Offer Novel Treatments For Genetic Disorders

According to a recent study, one of modern medicine’s biggest problems is managing debilitating inherited diseases. Victims and their families now have renewed hope thanks to the development of CRISPR technology and genetic research products over the past ten years. However, the security of these cutting-edge methods still causes grave alarm.

The study’s findings were released in the Science Advances publication.

CRISPR Technology: What Is It?

An innovative, risk-free technique that could one day be used to correct genetic faults has been reported by a group of scientists from the University of California, San Diego, which include professor Ethan Bier, research fellow Sitara Roy, and specialist Annabel Guichard. Their strategy, which utilizes the body’s natural DNA repair systems, paves the way for innovative gene therapy techniques with the potential to treat various genetic problems.

According to Guichard, the study’s senior author, the cell’s repair system can employ healthy variation to fix the flawed mutation after removing the mutant DNA. Even more effectively, this can be accomplished with a quick, non-lethal nick.

CRISPR Technology: How Does It Operate?

Working with fruit flies, the researchers created mutants that allowed the creation of pigments in their eyes to allow observation of such “homologous chromosome-templated repair,” or HTR. These mutants initially had all-white eyes. The same flies, however, displayed noticeable red spots across their eyes when the CRISPR components (a guide RNA and Cas9) were demonstrated. This was evidence that the cell’s DNA rebuild machinery had successfully reversed the mutation using the fully functioning DNA from the other chromosome.

They next put their new technique to the test using Cas9 variations called “nickases” that only targeted one strand of DNA rather than both. Surprisingly, the researchers discovered that these nicks also led to a high red-eye color restoration that was almost as good as that of healthy, normal (non-mutated) flies. They discovered that the nickase had a 50–70% success rate for repair, especially in comparison to merely 20–30% for dual–strand cutting Cas9, which also frequently produces mutations and hits other places throughout the genome.

In contrast to more conventional Cas9-based CRISPR edits, the team’s Nickas-based approach results in significantly fewer on- and off-target alterations, a critical aspect of their research. Additionally, they claim that multiple gradual, continuous nickase deliveries over several days may be more advantageous than a single, large delivery, as reported by ANI NEWS.