By: Gabriela del Amo
In today’s medical field, very few topics have been met with as much interest and argument as CRISPR. It has the kind of name that you would hear in a sci-fi movie, but the fact is that it exists: we can indeed change DNA. DNA is what makes up all living things and what differentiates all humans from one another. The CRISPR technology is a marvel that emerged in the early 2010s and now we are at a stage where it is used not only as a healing tool for inherited diseases but also to modify cancer therapies and is changing biotechnological treatments for generations to come. But what is CRISPR and why is it such a big deal?
The abbreviation CRISPR can be fully expanded to Clustered Regularly Interspaced Short Palindromic Repeats, isn’t that a mouthful! In simpler terms, it is a natural immune system of bacteria which fights against viruses. People managed to turn it into a precise gene editor only after they could figure out how to do it. The concept is fundamentally simple: reach the gene in the problematic DNA and either cut it out, replace it, or disable it.
The first step of the procedure is to have a gRNA, which stands for guide RNA that contains information of the target DNA sequence. This will come together with Cas9, which is an enzyme acting as a small pair of scissors. When the gRNA locates and captures its aim on the DNA, Cas9 cuts it off. Then the cell will repair the break by either shutting down the gene or pasting a new sequence. By using this method, the researchers can take out abnormal mutations or they can rectify the errors in the patient’s genetic blueprint.
Regarding the recipients, the impact CRISPR has on them is enormous. Victoria Gray can be mentioned as the first patient who was treated successfully with CRISPR for sickle cell disease, a type of blood disorder that is caused by a single abnormal gene and leads to a very painful condition. The therapy completely changed her bone marrow cells empowering them to produce normal red blood cells instead; and thus, she has not shown any symptoms for four years.
Regarding the recipients, the impact CRISPR has on them is enormous. Victoria Gray can be mentioned as the first patient who was treated successfully with CRISPR for sickle cell disease, a type of blood disorder that is caused by a single abnormal gene and leads to a very painful condition. The therapy completely changed her bone marrow cells empowering them to produce normal red blood cells instead; and thus, she has not shown any symptoms for four years.
Basically, one major advantage of CRISPR over traditional medical methods is the “one and done” hope that it holds for many. This means that instead of taking medications every day for the rest of their lives, patients may just need a single injection that deals with their disease at a molecular level. This will, no doubt, greatly enhance the quality of their lives, and thus, their physical saving, as too much medication intake usually has its own side effects.
For medical professionals and scientists, CRISPR has not only opened up new avenues of treatment, but also emerged as a powerful technology for the purpose of research. This tool can now enable them to devise more advanced disease models using gene editing in animals or cultured cells, where the employment of gene editing speeds up drug development as well as aids diagnosis of diseases more accurately. The use of CRISPR in the clinical sphere is a major step forward in the move towards tailor-made medicine that is genetically specific for each patient. The issue is not confined to the practical. It also opens up a deep moral discussion that has to be taken by healthcare professionals. Among the issues that should be dealt with are long-term safety and the consequences of inadvertently causing unprecedented problems as well as the very thorny issue of human DNA manipulation ethics. The representatives of the medical field are in a kind of hybrid age of bioengineering and information technology. Thus, in the future, they must have the required expertise to manage, regulate, and also address the public about CRISPR with great skill.
Looking at the future, CRISPR is developing in many areas of research. New methods, such as base editing and prime editing, can accomplish changes even more accurately, often not disrupting DNA at all. This reduces the incidents of unwanted changes and makes it possible to cure a wider variety of genetic diseases in humans. Besides, scientists have been finding some safer ways to transport CRISPR components into our bodies, using lipid nanoparticles or modified viruses. These advancements will allow the application of CRISPR to be more efficient for the different organs and cell types.
Visioning so far in the future, CRISPR may be the answer to day-to-day diseases such as the problems with the heart, the brain, high cholesterol, and not only unusual ones like those. At present, there are the moral doubts about modifying embryos, treatment accessibility, and long-term security without which CRISPR will not be able to move forward. Gene editing is continuously being developed, and so CRISPR is not any longer just a promising new technology but indeed it has become a complete game changer in modern medicine. Every time there is a new discovery, we are moving closer to the day when many inherited diseases are not only managed but also cured.
