Sickle No More - Is Sickle Cell Disease Now Curable?

Callum, Yasindhu & Aria, explore the ever-expanding world of gene therapy and gene editing. They investigate the gene therapy interventions for sickle cell patients and consider what this means for these patients going forward. Tune in to hear all about how gene therapy is already impacting lives!References:[1] Kanter, J., Walters, M.C., Krishnamurti, L., Mapara, M.Y., Kwiatkowski, J.L., Rifkin-Zenenberg, S., Aygun, B., Kasow, K.A., Pierciey, F.J. Jr., Bonner, M., Miller, A., Zhang, X., Lynch, J., Kim, D., Ribeil, J.A., Asmal, M., Goyal, S., Thompson, A.A., and Tisdale, J.F. (2022). Biologic and Clinical Efficacy of LentiGlobin for Sickle Cell Disease. N Engl J Med. 386(7), 617-628.[2] Singh, A., Irfan, H., Fatima, E., Nazir, Z., Verma, A., and Akilimali, A. (2024). Revolutionary breakthrough: FDA approves CASGEVY, the first CRISPR/Cas9 gene therapy for sickle cell disease. Ann Med Surg (Lond). 86(8), 4555-4559.[3] Ribeil, J.A., Hacein-Bey-Abina, S., Payen, E., Magnani, A., Semeraro, M., Magrin, E., Caccavelli, L., Neven, B., Bourget, P., El Nemer, W., Bartolucci, P., Weber, L., Puy, H., Meritet, J.F., Grevent, D., Beuzard, Y., Chrétien, S., Lefebvre, T., Ross, R.W., Negre, O., Veres, G., Sandler, L., Soni, S., de Montalembert, M., Blanche, S., Leboulch, P., and Cavazzana, M. (2017). Gene Therapy in a Patient with Sickle Cell Disease. N Engl J Med. 376(9), 848-855[4] Demirci, S., Uchida, N., and Tisdale, J.F. (2018). Gene therapy for sickle cell disease: An update. Cytotherapy. 20(7), 899-910.[5] Hoban M.D., Orkin, S.H., and Bauer, D.E. (2016). Genetic treatment of a molecular disorder: gene therapy approaches to sickle cell disease. Blood. 127(7), 839-848.[6] Orkin, S.H., and Bauer, D.E. (2019). Emerging Genetic Therapy for Sickle Cell Disease. Annu Rev Med. 70(1), 257-271.[7] Basu, A., Winn, A.N., Johnson, K.M., Jiao, B., Devine, B., Hankins, J.S., Arnold, S.D., Bender, M.A., and Ramsey, S.D. (2024). Gene Therapy Versus Common Care for Eligible Individuals With Sickle Cell Disease in the United States : A Cost-Effectiveness Analysis. Ann Intern Med. 177(2), 155-164.[8] Jones, R.J., Kassim, A.A., Brodsky, R.A., and DeBaun, M.R. (2025). Is allogeneic transplantation for sickle cell disease still relevant in the era of gene therapy?. Blood Adv. 9(4), 877-883.[9] Goyal, S., Tisdale, J.F., Schmidt, M., Kanter, J., Jaroscak, J., Whitney, D., Bitter, H., Gregory, P.D., Parsons, G., Foos, M., Yeri, A., Gioia, M., Voytek, S.B., Miller, A., Lynch, J., Colvin, R.A., and Bonner, M. (2022). Acute Myeloid Leukemia Case after Gene Therapy for Sickle Cell Disease. N Engl J Med. 386(2), 138-147.[10] Ball, J., Bradley, A., Le, A., Tisdale, J.F. and Uchida, N. Current and future treatments for sickle cell disease: From hematopoietic stem cell transplantation to in vivo gene therapy. (2025). Mol Ther. 33(5), 2172-2191.[11] Gray, V., Thomas, U., and Davies, K. (2024). Warrior Spirit: An Interview with Victoria Gray, Sickle Cell Pioneer. CRISPR J. 7(1), 5-11.

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