Antibiotic-Free Gene Vectors: A 25-Year Journey to Clinical Trials.

ABSTRACT: Until very recently, the major use, for gene therapy, specifically of linear or circular DNA, such as plasmids, was as ancillary products for viral vectors' production or as a genetic template for mRNA production. Thanks to targeted and more efficient physical or chemical delivery techniques and to the refinement of their structure, non-viral plasmid DNA are now under intensive consideration as pharmaceutical drugs. Plasmids traditionally carry an antibiotic resistance gene for providing the selection pressure necessary for maintenance in a bacterial host. Nearly a dozen different antibiotic-free gene vectors have now been developed and are currently assessed in preclinical assays and phase I/II clinical trials. Their reduced size leads to increased transfection efficiency and prolonged tr

SUBMITTER: Marie C 

PROVIDER: S-EPMC10970329 | biostudies-literature | 2024 Feb

REPOSITORIES: biostudies-literature