CRISPR-Cas9
Clustered regularly interspaced short palindromic repeats associated protein nine (CRISPR-Cas9) is a relatively new genome technology that is steadily gaining attention for its progression in genome editing and human embryonic gene editing in the media because of the ethical considerations that come with such an unknown area of science and medicine.
Advancements of the Medical Field through CRISPR-Cas9
Gene Therapy and Curing Genetic Diseases
“One of the key applications of CRISPR-Cas9 in medicine is in the study of gene function and disease mechanisms. By selectively modifying genes in human cells or model organisms, researchers can gain insights into the underlying causes of genetic diseases” (Arif Ansori). CRISPR has improved the understanding researchers have of genetic diseases like cystic fibrosis, hemophilia, and sickle cell disease, diseases that have plagued people in the U.S. for centuries. More progress can be made here using CRISPR to one day eliminate genetic diseases as a cause of suffering for future generations.
Discovering, Developing, and Screening New Drugs
"Gene editing is ready to have an immediate impact in real-world drug discovery and development…[as well as] large-scale functional screening with CRISPR-Cas9 which is simultaneously expanding and evolving" (Christof Fellman). CRISPR-Cas9 is extensively improving the methods in which drugs are being discovered, especially in tissue biomarking, and in how they are being tested/screened with technological models and computational tested therapies. All of these new drug discoveries and screenings are vastly improving the pharmaceutical world and making quicker advancements than ever before. This in turn can help generations to come for which diseases can be more quickly combated and can be done safely/effectively to prevent unnecessary deaths across the U.S.
Cancer Research and Therapies
"CRISPR-Cas9 has many potential uses in oncology, including creating cancer models, identifying targetable genes, evaluating for resistance mechanisms, and modulating efficacy of immunotherapies. The CRISPR/Cas9 system’s ability to create site-specific, highly efficient gene knockout makes it a desirable tool to address long-standing challenges, such as T-cell exhaustion and tumor microenvironment immunosuppression," (Dimitrios, Stefanoudakis). These systems have been effective thus far in completed and ongoing trials for clinical and preclinical phases of therapy. These pathways and avenues in cancer research using CRISPR-Cas9 have revolutionized the field to make the progress quicker and more applicable to the common cancers of today. This progress can help the next several generations until cancer can eventually become a problem of the past.
Success Has Already Begun
Trials for the use of CRISPR-Cas9 editing technology has already proved fruitful for several patients who have undergone clinical therapy. Victoria Gray is one of those patients. She suffered from a genetic disease called Sickle Cell Disease and had suffered from it for her whole life, being susceptible to other disease and was always too afraid to fly on planes for fear of being around so many other people who could get her and her family sick. After CRISPR gene therapy Victoria finally got to go on a plane to see her husband on a work trip saying "It was exciting! I had a window. And I got to look out the window and see the clouds and everything!" She is now thriving and doing well, and thus far her gene therapy has been a success in curing her genetic disease.
Leaders of the Future
"I'm very excited to see these results. Patients appear to be cured of their disease, which is simply remarkable." (Jennifer Doudna)
"I think it’s very unique because when you see all the field of just CRISPR biology, understanding the CRISPR-Cas systems in bacteria and archaea at the physiological level and even more at the mechanistic level, I mean this has been an incredible, explosion of knowledge and publications." (Emmanuelle Charpentier)
Success will Continue
Don't Let Progress Come to a Halt!
Some people debate that extensive limits and regulations need to be put in place on the use of this technology to prevent these things. While it is true to some degree that regulations need to be put into place surrounding this new technology and its uses, there is a certain point where it could negatively affect the progression it makes in the medical field for fear of a possible corruption of its use. The possibility of recreational use of this technology is farther off than people realize, and regulations are already being considered to be put in place to prevent such uses. A limit on the progress on then research use of stem cells for example could make the solutions to genetic diseases and even some cancers even farther from society’s future. Restrictions and regulations on the testing of CRISPR inventions, drugs, and processes for drug development, cancer research, and genetic diseases will stunt the growth and potential of this revolutionary technology.
Regulations are Already in Place
What many people don’t see are the regulations already put in place for clinical drug screening applications of this technology. All current uses of this technology, for things like drug development and gene therapy, are carefully thought/planned out and computer tested before it is ever tested on humans, meaning that there are already careful consideration and regulations put on the actual human uses for this technology.
Even though there are several ethical and regulatory concerns surrounding this new CRISPR technology, especially concerning embryonic editing, the main thing society should be focusing on is all the good that this technology can do for our medical future as a world of ever growing patients, with needs for advancements, some of which might only come through a discovery using CRISPR technologies.
LET CRISPR THRIVE!
Credits:
https://www.npr.org/sections/health-shots/2020/12/15/944184405/1st-patients-to-get-crispr-gene-editing-treatment-continue-to-thrive Ansori, Arif Nm, et al. “Application of CRISPR-Cas9 Genome Editing Technology in Various Fields: A Review.” Narra J, vol. 3, no. 2, 2023, pp. e184–e184, https://doi.org/10.52225/narra.v3i2.184. Brokowski, Carolyn, and Mazhar Adli. “CRISPR Ethics: Moral Considerations for Applications of a Powerful Tool.” Journal of Molecular Biology, vol. 431, no. 1, 2019, pp. 88–101, https://doi.org/10.1016/j.jmb.2018.05.044. Cetin, Busra, et al. “Gene and Cell Therapy of Human Genetic Diseases: Recent Advances and Future Directions.” Journal of Cellular and Molecular Medicine, vol. 28, no. 17, 2024, pp. e70056-n/a, https://doi.org/10.1111/jcmm.70056. Fellmann, Christof, et al. “Cornerstones of CRISPR–Cas in Drug Discovery and Therapy.” Nature Reviews. Drug Discovery, vol. 16, no. 2, 2017, pp. 89–100, https://doi.org/10.1038/nrd.2016.238. Ishino, Yoshizumi et al. “History of CRISPR-Cas from Encounter with a Mysterious Repeated Sequence to Genome Editing Technology.” Journal of bacteriology vol. 200,7 e00580-17. 12 Mar. 2018, doi:10.1128/JB.00580-17 Stefanoudakis, Dimitrios, et al. “The Potential Revolution of Cancer Treatment with CRISPR Technology.” Cancers, vol. 15, no. 6, 2023, p. 1813, https://doi.org/10.3390/cancers15061813.