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- Betibeglogene autotemcel for beta thalassemia: effectiveness and value : final evidence report1
- Chemistry, manufacturing, and control (CMC) information for human gene therapy investigational new drug applications (INDs)1
- Gene therapies for sickle cell disease: final evidence report1
- Gene therapy for hemophilia B and an update on gene therapy for hemophilia A: effectiveness and value : final evidence report1
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