Enhancing Nucleic Acid Delivery by siRNA and Pharmacological Agents

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.

Enhancing gene transfer into cells by utilizing siRNA or pharmacological agents. Understanding the desired target enhances and identifies the optimal pharmacological agent to use or gene to inhibit by siRNA.

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.

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, Director, The Cystic Fibrosis Biomarker Analysis and Assay Development RDP Core