STAT+: FDA chiefs offer roadmap to expand custom gene-editing treatments like Baby KJ’s
6.5
来源：
STAT
发布时间：
2025-11-12 20:00
摘要：
FDA officials have outlined a roadmap for the approval of personalized gene-editing treatments, inspired by the groundbreaking case of Baby KJ, who received a custom therapy for a rare liver disease. This initiative represents a significant advancement in biotechnology, requiring extensive collaboration among scientists and institutions. The treatment's development highlights the potential of gene editing to address unique genetic mutations, although the costs involved were substantial.
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关键证据
FDA officials detailed a roadmap for approving personalized gene-editing treatments.
The treatment for Baby KJ was a milestone in medicine.
The initiative required collaboration among numerous scientists and institutions.
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AI评分总结
FDA officials have outlined a roadmap for the approval of personalized gene-editing treatments, inspired by the groundbreaking case of Baby KJ, who received a custom therapy for a rare liver disease. This initiative represents a significant advancement in biotechnology, requiring extensive collaboration among scientists and institutions. The treatment's development highlights the potential of gene editing to address unique genetic mutations, although the costs involved were substantial.