Gene therapy showed a favorable safety profile, with no serious adverse events or evidence of genotoxicity. Efficacy was greater in younger children, in those without advanced marrow failure, and with higher doses of corrected CD34+ cells. However, as the included studies were phase I/II trials, the results should be interpreted cautiously; nevertheless, gene therapy appears to be a promising and safe treatment option in the early stages of the disease.
Reyes et al. (2026) studied this question.
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