Dossier
sirolimus
Coverage of sirolimus in the Nexus archive.
- STAT+: Drug meant to make gene therapy safer may also make it less effective
A new study finds that a medicine used to mitigate gene therapy side effects may also reduce its effectiveness. The trial tested a gene therapy for Dravet syndrome and used steroids and sirolimus to suppress immune responses. The results raise concerns about the balance between safety and efficacy in gene therapy.