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Rules, risks, and reality: rethinking gene therapy eligibility | Synapse
March 3, 2026
Open Access
Rules, risks, and reality: rethinking gene therapy eligibility
CK
Cécile Karsenty
AN
Alexander Ngwube
Baylor College of Medicine
Key Points
Gene therapy eligibility must be reconsidered to balance access and risk management.
The analysis reveals that many genetic disorders could benefit from broadening existing eligibility criteria.
Assessment using patient data explores the implications of expanding access to gene therapy for effective treatment.
The findings highlight the importance of understanding risks involved in gene therapy to guide future clinical applications.
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Karsenty et al. (Thu,) studied this question.
synapsesocial.com/papers/69a75e01c6e9836116a28582
https://doi.org/https://doi.org/10.1182/bloodadvances.2025018621
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