Paving the way for future gene therapies: A case study of scientific spillover from delandistrogene moxeparvovec.

ABSTRACT: Gene therapies have potential to improve outcomes of severe diseases after only a single administration. Novel therapies are continually being developed using knowledge gained from prior successes, a concept known as scientific spillover. Gene therapy advancement requires extensive development at each stage: preclinical work to create and evaluate vehicles for delivery of the therapy, design of clinical development programs, and establishment of a large-scale manufacturing process. Pioneering gene therapies are generating spillover as investigators confront myriad issues specific to this treatment modality. These include frameworks for construct engineering, dose evaluation, patient selection, outcome assessment, and safety monitoring. Consequently, the benefits of these therapies extend b

SUBMITTER: Asher D 

PROVIDER: S-EPMC10477757 | biostudies-literature | 2023 Sep

REPOSITORIES: biostudies-literature