Exth-65. Insertion of Microrna Target Sequences Into Retroviral Replicating Vectors Effectively Restricts Transgene Expression and Viral Replication in Human Hematopoietic Stem and Progenitor Cells
Abstract:Prodrug activator gene therapy with a retroviral replicating vector (RRV) has shown a highly favorable safety profile and long-term survival in early-phase trials for recurrent high-grade glioma. Overall endpoints were not met in a recent Phase 3 trial, but highly statistically significant survival was observed in prespecified patient subgroups as compared to randomized matched control patients receiving standard-of-care treatments, and further clinical evaluation is being focused on these subgroups. Additiona… Show more
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