This framework identifies candidates for genetic therapy in monogenic disorders, highlighting importance for patient outcomes.
The rapid expansion of genetic therapies for the treatment of monogenic disorders represents one of the most transformative developments in contemporary medicine. Given the novelty, significant risks, and high costs of these treatments, there is a need for a systematic approach to patient selection. We propose a patient-centered framework for identifying candidates for genetic therapy based on five key clinical questions and demonstrate its application through illustrative examples.
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Henein et al. (2026) studied this question.
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