In a recent groundbreaking feat in the medical field, CRISPR, a gene editing tool has been used to treat US cancer patients for the first time. This gene editing tool has been used for the first time on a trial basis to enhance the blood cells of two patients, one with sarcoma and another with multiple myeloma. The experimental research that is still under way at the University of Pennsylvania, works along the lines of genetically altering a person’s T cells so as to bring a change in them in a way that they attack and destroy cancer cells.
The landmark tool made its debut into the US clinical market with the treatment. Had it not been for the careful precautions that the team took to not muddle CRISPR’s introduction in the biomedical scene, the trial would have taken place at an earlier stage, soon after the team got their green flag from FDA to move forward with their concept in 2016. However, due to laxer rules of clinical registrations. China managed to conduct its first CRISPR-for-cancer trials in 2017, and has achieved a lead over other nations globally for the dominance of CRISPR. The complications in the precautions taken by the team can be owed to the history behind the Pennsylvania University Team and gene editing. At the turn of the century, the team put their bets and played a daring move when they treated an 18-year-old patient named Jesse Gelsinger using the gene therapy, which led to his death and a halt on the tides of promise that genetic tinkering was carrying with itself. This event is supposed to have set back the gene therapy by decades.
Now, let us address the basic question of what CRISPR is. It can be defined as a unique technology which is faster, cheaper and more versatile and precise than previously used techniques of editing DNA threads. It enables geneticists and other researchers in the field of medicine, to edit parts of the genome by removing, adding or altering sections of the sequence defined by DNA. The team has been working on the basics of the technology since a very long time and have come up with a basic layman concept behind the development of the technique. A lot of the primary bacteria function on a built-in gene editing system which is quite similar to the CRISPR-Cas9 system. This built-in system is used by them for responding to invasions by foreign pathogens and viruses, and acts like an immune system. CRISPR helps the bacteria to snip out little parts of the DNA of the foreign invading body and keep it behind, so as to recognise it and further help in defending the attacks of similar bodies. Therefore, a similar system was adopted by the scientists, to be used in other cells such as those of mica and humans.
Recently, numerous other gene editing technologies have been developed such as CRISPR-Cas systems, transcription activator-like effector nucleases (TALENs) and zinc-finger nucleases (ZFNs). These techniques provide the geneticists the advantage of controlling where in the genome the mutation would occur, as opposed to traditional techniques which also consumed a lot of time and were quite expensive. The technology has a lot of potential for proving to be a blessing when it comes to the treatment of a range of medical conditions including cancer, hepatitis B or even high cholesterol as all of these diseases involve having a genetic component.
The cancer study by the University of Pennsylvania is simply one of the numerous tests of medical treatments using CRISPR that are still in progress. Another example for the technology presented itself when a patient in Europe became the first person to be treated with CRISPR for an inherited disease, beta thalassemia. The future of the therapy is not very certain, as is the case with almost any and every groundbreaking experimental therapy, there are no guarantees. But often times, a leap of faith is all that is needed to work miracles and the hope that is placed by the medical community in the therapy, might just be sufficient to pull off a victorious battle against the deadliest of diseases.
Picture Courtesy- Genetic Engineering and Biotechnology News