CRISPR: Application Areas in Medicine
So, now for the first time in the history of medical science, we have a tool that can really allow gene editing in-Vitro and in-Vivo. It means we can edit genes in cell lines and then deliver these cells to the human body to treat health disorders. Or we can directly deliver #CRISPR to the body to carry out gene editing using injections, viral vectors, and more.
The possibilities here are almost endless. And it is incredible to see how fast the technology is maturing, and its first clinical application has begun within ten years of its discovery. So, the next ten years will be fascinating when we expect to see much broader clinical use of CRISPR.
If 2012 will be remembered as the year when CRISPR was discovered, then 2021 will be engraved in medical science history as the year when the first use of CRISPR began in clinical practice. In 2021, US #FDA gave its approval to start testing CRISPR to treat sickle cell anemia. Patients are given their own stem cells edited with CRISPR in the treatment, which results in significant benefits. As a result, researchers are expecting to be able to treat sickle cell anemia completely with the help of CRISPR. Of course, it will take a few years before therapy is approved for broader clinical use. Nonetheless, it is one of the most significant steps forward1.
Here are some of the areas of application of CRISPR in medicine:
Treat Genetic Disorders
It is perhaps the area of most significant interest. This is because so many genetic diseases are fatal. They cause years of disability and considerably reduced lifespan. Just take an example of blood disorders like sickle cell disease and thalassemia, or consider Duchenne muscular dystrophy1,2.
Here the treatment approaches could be quite different. For example, in the case of sickle cell #anemia, researchers use stem cells extracted from the patient’s body, edit them using CRISPR, and then reintroduce them into the body. These healthy stem cells start producing healthy blood cells, thus providing relief.
In the case of #Duchenne muscular dystrophy, doctors would need to directly inject CRISPR into muscles to edit genes causing the dystrophy or use some vector like adenovirus to carry out more extensive gene editing.
Above are just examples of how CRISPR may work in different ways to treat genetic disorders. Of course, here, possibilities are numerous. Once researchers have found a way to treat these disorders, they may turn their attention to type 1 diabetes, autoimmune diseases, and even metabolic disorders. CRISPR may even have a role in neurodegenerative disorders.
Treat cancers
The traditional approach to #cancer treatment is #chemotherapy, along with #radiotherapy and surgical removal. However, these approaches are pretty harmful to #health, cause many adverse effects, and fail to eliminate cancer cells completely.
Thus, one of the newer approaches is immune therapy, which stimulates certain immune cells, making them more active against cancer cells. However, cancer cells develop resistance over time and find a way to escape immunity. Therefore, researchers are testing CRISPR for modulating #immune cells like T-cells to more effectively identify cancer cells and thus help initiate immune response3,4.
Such kind of treatment would have fewer side effects and may result in a complete cure for different cancers.
Treat infectious diseases
Yes, CRISPR can one day help treat some of the most complex infections. Just take the example of #HIV; it can insert its genetic material into the human genome and then become inactive when exposed to highly active antiretroviral therapy. Thus, a person has to keep taking antiretroviral drugs lifelong. However, CRISPR can identify and delete the viral genetic material, providing HIV cure2.
Or take the example of malaria, CRISPR may help produce mosquitoes that do not carry malaria, and these mosquitoes can be propagated. This will slowly eliminate malaria, as all mosquitoes would ultimately become resistant to the parasite2.
Another good example of how it may help fight infections could be genetically modified bacteriophages. Bacteriophages are viruses that can attack and kill specific kinds of bacteria. However, CRISPR can help modify these viruses to start targeting different bacteria. This may even help overcome antibiotic-resistant infections3.
Diagnostics
Another significant application of CRISPR in medicine could be in the area of #diagnostics. CRISPR can not only diagnose specific infections but may also help identify the exact strain causing the #infection. Moreover, CRISPR-based diagnostic tools can be pretty sensitive. Researchers have already created some diagnostic tools, like those to detect #Covid19. Since such tools are part of in-vitro diagnostics, they can be more readily introduced for broader use in medicine.
Another exciting area where CRISPR can be helpful is in identifying genetic mutations that occur during the course of life, thus causing various health disorders. For example, genome-wide association studies (GWAS) have identified many pathogenic single nucleotide polymorphisms (SNPs) related to different diseases. It appears that CRISPR can help identify these mutations quickly and effectively. Furthermore, SNPs play a role in the development of almost 50% of all diseases; thus, CRISPR can change diagnostics forever.
Conclusion
To sum up, it is vital to understand that CRISPR is not just a tool to treat human genetic diseases. It has a much broader scope. It can even help overcome other chronic ailments, treat cancers, and treat chronic infections. CRISPR can help diagnose and treat almost all kinds of diseases. More importantly, it may help treat the root cause, thus providing permanent relief for many health conditions.
References
1. Sanders R, relations| M. FDA approves first test of CRISPR to correct genetic defect causing sickle cell disease. Berkeley News. Published March 30, 2021. Accessed October 20, 2022. https://news.berkeley.edu/2021/03/30/fda-approves-first-test-of-crispr-to-correct-genetic-defect-causing-sickle-cell-disease/
2. Mahmoudian-sani MR, Farnoosh G, Mahdavinezhad A, Saidijam M. CRISPR genome editing and its medical applications. Biotechnology & Biotechnological Equipment. 2018;32(2):286–292. doi:10.1080/13102818.2017.1406823
3. CRISPR Clinical Trials: A 2022 Update. Innovative Genomics Institute (IGI). Accessed October 20, 2022. https://innovativegenomics.org/news/crispr-clinical-trials-2022/
4. Tiruneh G/Medhin M, Chekol Abebe E, Sisay T, Berhane N, Bekele T, Asmamaw Dejenie T. Current Applications and Future Perspectives of CRISPR-Cas9 for the Treatment of Lung Cancer. Biologics. 2021;15:199–204. doi:10.2147/BTT.S310312
