Copy, Cut, Paste: Who Gets to Rewrite Their Genes

Authored by: Zianna Odogwu

Art by: Alex Song

With the rise of revolutionary technology and medicine, large language models and new phones aren't the only technologies reshaping everyday life. Particularly, human life is significantly changing for sickle cell disease patients who may have a renewed chance through CRISPR gene editing with continued research and scientific testing. Sickle cell anemia is a variant of hereditary blood disorders that comprise sickle cell disease. While sickle cell disease is caused by a general mutation in the HBB gene, sickle cell anemia is specifically attributed to a genetic mutation in which both copies of the HBB gene produce hemoglobin SS, the most common and severe form of SCD. The mutation causes red blood cells that are normally round and flexible to become stiff and crescent-shaped, which can lead to life-threatening sickle cell crises as crucial blood flow is slowed and blocked [1]. Historically, treatment options for sickle cell patients have been limited. Non-invasive options include infection-preventing antibiotics and blood transfusions that provide healthy red blood cells to mitigate crises. In addition, hydroxyurea, L-glutamine, and Crizanlizumab are used as disease-modifying agents to make blood cells less likely to adopt a mutated form. Outside preventative therapies, few sickle cell patients can opt for bone marrow transplantation or Hematopoietic Stem Cell Transplantation (HCST), through which patients are matched with a healthy donor that provides bone marrow [2]. However, for those who cannot afford these costly therapies or elect not to participate, the burden of sickle cell anemia becomes substantially greater.

In late 2024, pharmaceutical companies CRISPR Therapeutics and Vertex released a CRISPR/Cas9–based treatment for sickle cell disease by leveraging gene-editing abilities to precisely target and modify the mutated β-globin gene and/or reactivate fetal hemoglobin production, offering a permanent fix contrasting current treatments that solely focus on managing symptoms [2]. Clustered Regularly Interspaced Palindromic Repeats (CRISPR), a natural bacterial defense against phages, captures viral DNA in "CRISPR arrays," enabling enzymes like Cas9 and guide RNA to cut matching DNA [3]. Its use in disease-causing mutations began in MIT labs within mice carrying the human tyrosinaemia mutation, accelerating the CRISPR-research race. Since the early 2010s, CRISPR-based treatments have come a long way in addressing substantial ethical, safety, and practical concerns to expand its use in clinical and scientific research [4]. In March of 2021, the FDA approved Casgevy as the first therapy in clinical medicine to use CRISPR-Cas9 for the treatment of sickle cell disease. The highly successful initial trial yielded "significant restoration of functional hemoglobin, reduced sickle cell-associated pain, and slowed disease progression," allowing the therapy's official approval in patients aged 12 and older [2]. Casgevy has already begun making its impact, particularly in the life of Victoria Gray, a sickle cell patient who no longer suffers from sickle cell-related pain and attacks due to treatment she underwent in 2021. Gray has emphasized how the treatment transformed her physical and mental health, citing that common colds are no longer life-threatening, she has fully returned to school, and now lives a normal life [5].

Gray is one of many sickle cell patients of African descent. More than 90% of patients affected by sickle cell disease are of non-Hispanic Black or African American lineage [6]. Given the majority Black patient population, examining the broader social contexts of this scientific discovery highlights how Casgevy goes beyond simple medical innovation to a harbinger of health equitable biomedical research. With sickle cell's history as a "black disease," used to discriminate and clinically profile Black patients, Casgevy symbolizes a push through stigma and bias, manifesting in public perception through educational materials exclusively featuring darker-skinned individuals, racial profiling, and marginalizing sickle cell patients as "excessively stoic and chronically drug-seeking." Consequently, achieving quality biomedical research in a heavily stigmatized and racialized disease has been tumultuous for scientists who have historically battled extensive public backlash in advocating for increased visibility of sickle cell research [7]. Barriers such as a general distrust of biomedical institutions in Black populations have further contributed to delayed clinical trials. For these reasons, medical and scientific professionals have celebrated how treatment has served in addressing long-standing health inequities that have largely jeopardized both the health of sickle cell patients and the health of Black and Brown populations [8].

As an African-American myself with insight into how sickle cell anemia has impacted Black populations around me, I am hopeful that Casgevy represents a meaningful possibility for Black patients who have long been devastated by a costly disease amid historical underinvestment in Black health. With this in mind, it's imperative to consider the nuances behind the high cost of treatment that challenge these advancements as a symbol of health equity. Currently, Casgevy is priced at 2.2 million US dollars per patient, which for any average patient and most private insurance companies is an unattainable sum, making the treatment solely a pipe dream for most [9]. Cost is particularly a barrier for a majority Black patient population, as the median income for African Americans stands at 55,000 to 56,700 US dollars [10]. Casgevy still represents a step in the right direction, but acknowledging the paradox reveals that a treatment born from addressing a historically marginalized "Black disease" advances health equity in principle, while in practice deepening inequity by remaining accessible primarily to those in wealthier nations.

References:

1. Cleveland Clinic. (2023, August 3). Sickle Cell Disease. Cleveland Clinic. https://my.clevelandclinic.org/health/diseases/12100-sickle-cell-disease

2. Tariq H, Khurshid F, Khan MH, Dilshad A, Zain A, Rasool W, Jawaid A, Kunwar D, Khanduja S, Akbar A. CRISPR/Cas9 in the treatment of sickle cell disease (SCD) and its comparison with traditional treatment approaches: a review. Ann Med Surg (Lond). 2024 Aug 14;86(10):5938-5946. doi: 10.1097/MS9.0000000000002478. PMID: 39359808; PMCID: PMC11444630.

3. Fuguo Jiang, Jennifer A. Doudna. 2017. CRISPR–Cas9 Structures and Mechanisms. Annual Review Biophysics. 46:505-529. https://doi.org/10.1146/annurev-biophys-062215-010822

4. Ledford, H. (2015). CRISPR, the disruptor. Nature, 522(7554).

5. Stein, R. (2021, December 31). First sickle cell patient treated with CRISPR gene-editing still thriving. NPR. Retrieved February 26, 2026, from  https://www.npr.org/sections/health-shots/2021/12/31/1067400512/first-sickle-cell-patient-treated-with-crispr-gene-editing-still-thriving

6. Centers for Disease Control and Prevention. (2024, May 15). Data and Statistics on Sickle Cell Disease. CDC. https://www.cdc.gov/sickle-cell/data/index.html

7. Benjamin, R. (2014). Organized ambivalence: when sickle cell disease and stem cell research converge. In Genetics and Global Public Health (pp. 141-157). Routledge.

8. Santiago J. Molina, Melissa Creary, The racial politics of visibility and equity in genome-editing therapies for sickle cell disease, Social Science & Medicine,  Volume 383, 2025, 118452, SSN 0277-9536, https://doi.org/10.1016/j.socscimed.2025.118452. (https://www.sciencedirect.com/science/article/pii/S027795362500783X)

9. King, M. D. (2026, January 29). How Income Varies by Race and Geography. U.S. Census Bureau. https://www.census.gov/library/stories/2026/01/household-income-by-race-and-state.html