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CRISPR Base Editors in Clinical Trials: Balancing Precision with Off-Target Surveillance
碱基编辑器进入临床试验：在精准治疗与脱靶监测之间取得平衡
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Unlike traditional CRISPR-Cas9, base editors chemically convert one DNA nucleotide into another without inducing double-strand breaks.
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Recent Phase I trials for sickle-cell disease use adenine base editors to correct the HBB gene mutation directly in autologous hematopoietic stem cells.
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Yet whole-genome sequencing of edited cell populations reveals low-frequency off-target edits—especially in repetitive genomic regions.
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Clinicians now mandate orthogonal detection methods like Digenome-seq and targeted long-read sequencing before patient reinfusion.
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Regulatory submissions must include off-target risk scores derived from cell-type-specific chromatin accessibility maps and RNA expression profiles.
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Ongoing trials treat editing not as a one-time intervention but as a monitored therapeutic process requiring longitudinal genomic follow-up.