I finished my degree in Sociology with a minor in History, and since I work at a University that will let me receive more education for free, I have decided to pursue a new degree in Information Technology. I am currently taking an entry-level tech class where I am exploring different types of technology and how it interacts with various fields, environments, and workplaces. This course is surprisingly heavy when it comes to shorter papers (2-5 pages), and so I will be uploading new content to this blog that will have a technology vibe to it.
Recent breakthroughs in gene-editing technology have opened new possibilities for treating genetic disorders. In late 2023, the FDA approved two CRIPSR-based gene therapies – Casgevy and Lyfgenia – for sickle cell disease, marking a significant milestone in precision medicine. These therapies work by modifying patients’ blood stem cells to increase fetal hemoglobin, which prevents the sickling of red blood cells. Sickle cell disease affects approximately 100,000 individuals in the United State and is associated with severe pain, organ damage and reduced life expectancy. The potential benefits of CRISPR-based therapies include improved quality of life, fewer complications, and reduced healthcare costs (U.S. Food and Drug Administration, 2023). Traditional treatments such as blood transfusions and bone marrow transplants are costly, risky, and not curative. In contrast, CRISPR therapy offers the possibility of a one-time, curative solution. (Saionz, 2025).
The core innovation behind CRIPSR-based gene therapy for sickle cell disease (SCD) lies in its ability to precisely edit a patient’s DNA to correct or bypass the genetic defect responsible for the condition. CRIPSR/Cas9 is a groundbreaking gene-editing tool that enables scientists to target and modify specific genes with remarkable accuracy. In the case of SCD, the therapy focuses on editing blood stem cells to increase the production of fetal hemoglobin (HbF) – a from of hemoglobin that does not sickle and can functionally replace the defective adult hemoglobin (Azar, 2024). Stem cells are harvested from the patient’s bone marrow and edited using CRISPR/Cas9 to disrupt the BCL11A gene, which normally suppresses HbF production. The modified stem cells are then re-infused into the patient, where they begin generating healthy red blood cells containing fetal hemoglobin (Harvard Medical School, 2025). This approach offers the potential for a one-time, curative treatment, rather than lifelong disease management.
Sickle cell disease is a debilitating blood disorder, and CRISPR-based gene therapy offers new hope for patients who have long suffered from chronic pain, anemia, organ damage, stroke, and reduced life expectancy. By addressing the root genetic cause, this therapy has the potential to dramatically improve quality of life. Beyond sickle cell disease, CRISPR technology holds promise for treating other inherited blood disorders, such as beta-thalassemia, which also results from faulty hemoglobin production. The broader category of hemoglobinopathies presents numerous opportunities for gene-editing research. Additionally, scientists are exploring whether CRISPR could be adapted to target certain cancers or rare genetic conditions like muscular dystrophy and cystic fibrosis (Saionz, 2025)
For patients undergoing CRISPR therapy, the immediate benefits include a reduction in painful episodes caused by blocked blood flow from sickled cells. Many report decreased fatigue, fewer hospital visits, and an increased ability to participate in daily activities. These improvements also benefit healthcare systems by reducing emergency room visits and streamlining treatment into one-time intervention rather than ongoing management.
As with any groundbreaking medical innovation, CRISPR-based gene therapy raises important concerns about access, affordability, and responsible use. Sickle cell disease disproportionately affects individuals of African descent, particularly those in low-income communities. These populations have historically faced systematic barriers to healthcare, and there is growing concern that advanced treatments like CRISPR may not be equitably distributed (Molteni, 2023). The launch prices for the FDA-approved therapies – Casgevy and Lyfgenia – are staggering, making them inaccessible to most patients without substantial insurance coverage or government assistance. In response, the U.S. Centers for Medicare and Medicaid Services introduced the Cell and Gene Therapy Access Model, which allows states to negotiate outcome-based payment agreements with manufacturers. This initiative acknowledges that many individuals who need these therapies are also among those least able to afford them. (Cohen, 2025).
While CRISPR technology is revolutionizing healthcare, it also has profound implications across other fields – particularly agriculture. Scientists have used CRISPR to enhance crop yield, nutritional value, and resistance to pests, diseases, and environmental stress. It has been applied to boost levels of vitamins and minerals, improve taste and shelf life, and reduce allergens in food. For example, susceptibility genes in crops like rice and wheat have been knocked out to make them resistant to fungal and bacterial infections. Instead of relying on chemical pesticides, crops can be edited to naturally deter pests, increasing insect resistance. CRISPR also enables crops to survive with less water or thrive in extreme climates such as heat or cold (Atimango, 2024). Many of these foods are already part of our food system, often labeled as GMOs. However, regulatory approaches vary: some countries treat CRISPR-edited foods like traditional GMOs, while others – such as the United States – do not, provided no foreign DNA is introduced. Public acceptance of CRISPR-edited foods varies widely, and education will be key to building trust and understanding.
CRIPSR-based gene therapy represents a groundbreaking advancement in the treatment of sickle cell disease, offering the possibility of a one-time cure for the condition that has long been managed through costly and limited interventions. While the scientific and clinical benefits are profound, the technology also raises important questions about equitable access, affordability, and responsible use. Beyond healthcare, CRISPR’s potential extends into agriculture and other fields, demonstrating its versatility and transformative power. As research and policy continue to evolve, it will be essential to ensure that these innovations are both effective and accessible to those who need them most.
Resources:Atimango, Alice. O (2024). Genome Editing in Food and Agriculture. Trends in Food Science & Technology. https://doi.org/10.1016/S0958-1669(24)00063-6
Azar, S. (2024, June 10). CRISPR gene therapy for sickle cell disease. Mass General Brigham. https://www.massgeneralbrigham.org/en/about/newsroom/articles/gene-therapy-for-sickle-cell-disease
Cohen, J. (2025, August 2). Novel access model for sickle cell disease gene therapy could be template. Forbes. https://www.forbes.com/sites/joshuacohen/2025/08/02/novel-access-model-for-sickle-cell-disease-gene-therapy-could-be-template/
Harvard Medical School. (2025, February 20). Creating the world’s first CRISPR medicine, for sickle cell disease. https://hms.harvard.edu/news/creating-worlds-first-crispr-medicine-sickle-cell-diseaseSaionz, A. (2025, July 1). Cell & gene therapies in 2025: Breakthroughs, challenges, and the path to accessible innovation.
Molteni, M. (2023, March 7). CRISPR cures for sickle cell disease raise equity concerns. STAT. https://www.statnews.com/2023/03/07/crispr-sickle-cell-access/
PharmaBoardroom. https://pharmaboardroom.com/articles/cell-gene-therapies-in-2025-breakthroughs-challenges-and-the-path-to-accessible-innovation/
U.S. Food and Drug Administration. (2023, December 8). FDA approves first gene therapies to treat patients with sickle cell disease [Press release]. https://www.fda.gov/news-events/press-announcements/fda-approves-first-gene-therapies-treat-patients-sickle-cell-disease
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