Clustered Regularly Interspaced Short Palindromic Repeats or “CRISPR” for short is a technology being developed to edit genes. The technology utilizes the immune system found in microbes to find and dispose of specific viruses or other harmful invaders.
The technology has constantly improved ever since its first discovery in 1987 by Japanese scientist Yoshizumi Ishino in Osaka, Japan and has made some recent improvements.
In the year of 2024, advancements in the field of vivo genome editing have developed at a steady pace. In March 2024, Tune Therapeutics shared that they are developing TUNE-401, a potential cure for the chronic hepatitis B virus that will work as a “one-and-done” treatment. So far, the preclinical data for it has shown an almost “complete repression” of the RNA responsible for HBV and Tune has received approval to begin clinical trials.
Eligo Bioscience has also made major headway with CRISPR technology in the field of microbiome editing. In July 2024 they discovered with lab testing that it was possible to target specific bacteria within the gut biome of a living mouse. Their findings, later published in Nature, the world’s leading multidisciplinary science journal, opened up the possibility to selectively modify bacterial genes that cause or influence certain therapies used to combat diseases.
While CRISPR is not the only gene-editing technology available to the scientific community at this point in time, it is easier to program. Most gene-editing technologies make use of a single protein strand in order to alter their intended targets through a change in the peptide sequence, however CRISPR encodes their sequence into a ribonucleic acid or RNA, with the RNA acting as a “guide” that makes the process much smoother.
However, while there have been many recent developments, there are still many hurdles left in order to properly introduce this technology into the medical field and get a full grasp on its many potential uses.
“Despite the clinical and preclinical successes of CRISPR, further innovations in delivery systems are pivotal.” – Jo Qian, University of North Georgia biology professor and geneticist
As Qian detailed, the treatment itself is “nothing like swallowing a Tylenol pill to reduce a fever,” with the main concern in advancement today still revolving around effective delivery. She continued that being able to efficiently target tissues and cells is a main goal to achieve with other recent advancements such as biodegradable nanoparticles for lung-targeted delivery and acid-degradable lipid nanoparticles to cross the blood brain barrier to show “great promise.”
