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For instance, Casgevy, based on the CRISPR/Cas9 gene-editing system, was the first successful attempt at disease treatment, promising a one-time treatment for a lifelong effect. However, the complexity of clinical applications and the difficulty of target selection in gene editing have led to long treatment cycles and high costs

Gasser M, Lenglet S, Bararpour N, Sajic T, Wiskott K, Augsburger M, et al