Martina Rossi Science Reviews - Biology, 2023, 4(3), 15-18
Acknowledging the Approval of World-First Gene
Therapy for Sickle Cell Disease Through CRISPR-
Mediated Gene Editing
Martina Rossi
, PhD
Independent Researcher, Strasbourg, France;
1. Pflaum, C. (2023). FDA Approves First Gene Therapies to Treat Patients with Sickle Cell Disease”.
FDA News Release, 8 December. Available at:
2. Rossi, M. (2023). Advancements and Challenges in Gene Therapy Approaches for Sickle Cell
Disease: A Comprehensive Review”, SciRevs Biology, 2(3), p. 3.
3. Thein, S. L. (2017). Genetic Basis and Genetic Modifiers of β-Thalassemia and Sickle Cell Disease,
Adv. Exp. Med. Biol. PMID:29127676.
4. Germino-Watnick, P., Hinds, M., Le, A., Chu, R., Liu, X., & Uchida, N. (2022). Hematopoietic Stem
Cell Gene-Addition/Editing Therapy in Sickle Cell Disease”, Cells, 11(11), 1843. PMCID: PMC9180595. PMID: 35681538.
5. Sankaran, V. G., & Orkin, S. H. (2013). The Switch from Fetal to Adult Haemoglobin, Cold Spring
Harb Perspect Med, 3(1), a011643. PMCID:
PMC3530042. PMID: 23209159.
6. Wong, C. (2023). UK first to approve CRISPR treatment for diseases: what you need to kno, Nature.