Phase 1 Trial of Crispr-Cas9 Gene Editing Targeting Angptl3
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Crispr Gene EditingClinical TrialCardiovascular Health
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Crispr Gene Editing
Clinical Trial
Cardiovascular Health
A phase 1 trial of CRISPR-Cas9 gene editing targeting ANGPTL3 showed promising results with no dose-limiting toxic effects and few adverse events, sparking discussion on the safety and potential of this therapeutic approach.
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> Editing of ANGPTL3 was associated with few adverse events and resulted in reductions from baseline in ANGPTL3 levels.
Two out of 15 having adverse advents seems pretty high, especially since one died. The supplemental material rules this as an unrelated death (https://www.nejm.org/doi/suppl/10.1056/NEJMoa2511778/suppl_f...).
Definitely seems promising for a larger-scale trial, but it seems weird to make such a strong conclusion about adverse reactions.