INTRODUCTION:
UCLA researchers in the Departments of Integrative Biology & Physiology and Pathology & Laboratory Medicine have developed a novel construct for targeting therapeutic biological molecules specifically to diseased tissue, using immune cells as a delivery system.
BACKGROUND:
Drug delivery is the process of administering a pharmaceutical agent for a therapeutic benefit to patients. Routes of delivery, delivery vehicles, and therapeutic packaging differ between drug delivery systems, and may be individually curated to increase efficacy and proper drug targeting. However, conventional methods of pharmaceutical delivery via systemic administration exposes the whole body to a drug, which often causes undesirable, off-target side effects. Even once the drug reaches its desired tissue, the dosage may not be therapeutically maximal. There is significant interest in the development of novel drug delivery systems that target therapeutic molecules directly to diseased tissue, to increase the efficiency and safety of therapeutics.
INNOVATION:
UCLA researchers have created a DNA construct that is targeted in vivo to deliver a therapeutic, biological molecule specifically to sites of disease by immune cells. The immune cells are engineered to robustly produce the therapeutic molecule, and migrate to the diseased tissue by endogenous processes of inflammatory immune response. This system takes advantage of the natural mechanisms of immune cells to target biologicals directly to affected tissue. The targeted construct will maximally mitigate inflammation and promote tissue growth and repair, without dangerous off-target effects. Ultimately, this system allows for a safer, more efficient delivery of therapeutic compounds.
POTENTIAL APPLICATIONS:
• Delivery of therapeutic, biological molecules directly to diseased tissue
ADVANTAGES:
• Therapeutic molecule delivery increased specifically in damaged tissue
• Avoids off-target drug effects
• Safer, more efficient therapeutic delivery compared to systemic drug administration
DEVELOPMENT-TO-DATE:
This technology has been tested both in vitro and in vivo.