Gene augmentation has been the paradigm in the majority of gene therapy protocols but in recent years the potential of repairing the mutated gene in situ by targeted gene correction has become a reality. In fact, targeted gene repair has many advantages over conventional replacement strategies, notably the possibility to treat dominant as well as recessive disorders, and the small molecular size of the pharmacologically active agent. Chimeric RNA/DNA oligonucleotides, small fragment homologous replacement, as well as triplex-forming and single-stranded oligonucleotides are all examples of the growing armamentarium for gene repair, and are the subject of this review. In addition, we have also included a discussion of the reawakened Sleeping Beauty (SB) transposon system as a novel non-viral gene replacement strategy.
展开▼
