A novel non‑viral, non‑lipid delivery system enables safe transfer of large genetic payloads, avoiding key limits of current gene therapies and improving uptake using targeted siRNA or drug-based enhancers.

A novel immunocapture procedure identified 523 unique proteins that interfere with gene transfer.  Two methods, RNAi and small molecules, have been identified to enhance gene transfer by modulating specific targeted proteins that prevent the nucleic acid from entering that part of the cell.  RNAi molecules were identified that target and knock down specific cellular proteins that negatively impact the uptake of the nucleic acid delivery vehicle.  Results demonstrate RNAi decreases specific cellular proteins, reducing their impact on the downstream transfer of nucleic acids.  The second, small molecule approach, also targets proteins that interfere with gene transfer.

Cystic Fibrosis

Current gene therapy approaches rely largely on viral vectors or lipid nanoparticles which face challenges with immunogenicity, limited payload capacity, safety, and complex manufacturing. This non-viral, non-lipid delivery directly addresses these challenges.

Gene transfer is defined as a technique to efficiently and stably introduce foreign genes into the genome of target cells. Gene transfer technologies were originally developed as a research tool for investigating gene expression and function. However, new focus and development on new gene transfer technologies is enabling an expansion of new applications.

Assem Ziady, PhD