Review article. Strategies for in vivo targeted gene silencing

Background: Small interfering RNA (siRNA) has attracted extensive attention showing significant promise for the study, diagnosis, and treatment of human disease. However, the specific and efficient delivery of siRNA into cells in vivo remains a great challenge. Targeted modification of siRNA, viral nanoparticle-based vectors, targeted multifunctional/multistage nanosystems, combining ultrasound-targeted microbubble destruction, and tumor targeting in an all-in-one system, provides a useful multimodal approach in targeted delivery.

Objective: We provided an overview of different strategies for siRNA delivery including direct modification of siRNA, nanoparticles and viral vectors.

Methods: We conducted a search of standard database. Relevant primary and summary resources were identified and abstracted. A summary of strategies for in vivo targeted gene silencing was produced.

Results: A list of strategies for gene-targeted delivery in vivo was summarized, including target cells, target genes, target legends, and disease model for each strategy. An overview of strategies for siRNA delivery aimed at in vivo targeted gene silencing was presented.

Conclusions: Integration of the advantages of viral or nonviral vectors into gene silencing could have profound impacts on biomedical research. Recent progress is pointing at answers.