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Since AAVs have been found to integrate within DSBs with high frequency [152], the coupling of CRISPR-Cas9 with AAVs as the delivery vector must be carefully evaluated before clinical translation [58, 153]

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First, the historical absence of a globally unified definition of rare diseases constrained our study scope to the top ten rare diseases with the highest R&D activity, which may not fully represent the broader spectrum of rare disease research

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