Methodologies to investigate intracellular barriers for nucleic acid delivery in non-viral gene therapy

A Triple-Fluorophore-Labeled Nucleic Acid pH Nanosensor to Investigate Non-viral Gene Delivery.

There is a need for new tools to better quantify intracellular delivery barriers in high-throughput and high-content ways. Here, we synthesized a triple-fluorophore-labeled nucleic acid pH nanosensor for measuring intracellular pH of exogenous DNA at specific time points in a high-throughput manner by flow cytometry following non-viral transfection. By including two pH-sensitive fluorophores an...

Barriers and recent advances in non-viral vectors targeting the lungs for cystic fibrosis gene therapy

Cystic fibrosis (CF) is an autosomal recessive disorder caused by mutations in CFTR genes that affect chloride ion channel. The CF is a good nominee for gene therapy as the asymptomatic carriers are phenotypically normal, and the desired cells are accessible for vector delivery. Gene therapy shows promising effects involving the correction of gene or replacement of the mutant gene with the func...

Endosomal Escape Pathways for Non-Viral Nucleic Acid Delivery Systems

Non-Viral Delivery Systems in Gene Therapy

Recent advances in molecular biology combined with the culmination of the Human Ge‐ nome Project [1] have provided a genetic understanding of cellular processes and disease pathogenesis; numerous genes involved in disease and cellular processes have been identi‐ fied as targets for therapeutic approaches. In addition, the development of high-throughput screening techniques (e.g., cDNA microarra...

Microglial Barriers to Viral Gene Delivery

In this issue of Neuron, Tufail et al. present an underlying mechanism for microglia-mediated elimination of virally transduced cells in the central nervous system. These findings could contribute to the development of improved gene therapies for various neurological disorders by exploring why microglia destroy viable cells following viral infection.