Safety and Efficacy Studies of CRISPR-Cas9 Treatment of Sickle Cell Disease Highlights Disease-Specific Responses

Frati, Giacomo, et al. « Safety and Efficacy Studies of CRISPR-Cas9 Treatment of Sickle Cell Disease Highlights Disease-Specific Responses ». Molecular Therapy: The Journal of the American Society of Gene Therapy, vol. 32, n^o 12, décembre 2024, p. 4337‑52. https://doi.org/10.1016/j.ymthe.2024.07.015