Nanoparticles that boost gene editing to treat solid tumors
Multiplexed nanoparticle delivery to increase CRISPR/Cas gene editing for enhanced cancer therapy
This project uses tailor-made nanoparticles to deliver gene-editing tools into hard-to-reach solid tumors to help people with liver and lung cancers.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Ut Southwestern Medical Center NIH-funded |
| Lab location | 1 site (Dallas, United States) |
| Project ID | NIH-11294164 on NIH RePORTER |
What this research studies
From my perspective as a patient, the team is combining tiny lipid particles that travel through the blood with two genetic tools: one that temporarily softens the tumor environment (FAK siRNA) and one that performs permanent gene edits (Cas9 mRNA plus guide RNA). They plan to give these particles by IV so they can reach tumors in the liver and lung using a platform called SORT LNPs. The idea is that weakening the tumor’s stiff tissue will let the particles get in better and allow CRISPR to more effectively delete cancer-related genes like PD-L1. Most of the work is preclinical using tumor models to test delivery, editing efficiency, and safety before any patient trials.
Who could benefit from this research
Good fit: Ideal candidates for future trials would be people with solid hepatocellular carcinoma (liver cancer) or non-small cell lung cancer who are eligible for systemic IV nanoparticle therapies.
Not a fit: People with blood cancers, tumors not reachable by IV nanoparticles, or those who need immediate standard-of-care treatment may not benefit from this approach.
Why it matters
Potential benefit: If successful, this could enable permanent inactivation of immune-suppressing or cancer-driving genes in solid tumors and improve responses to therapy.
How similar studies have performed: Lipid nanoparticle delivery and in vivo CRISPR editing have shown early clinical promise in liver-targeted applications, but combining ECM-targeting siRNA with CRISPR in a single particle for solid tumors is largely new and experimental.
Where this research is happening
Dallas, United States
- Ut Southwestern Medical Center — Dallas, United States (Active)
Researchers
- Principal investigator: Siegwart, Daniel John — Ut Southwestern Medical Center
- Study coordinator: Siegwart, Daniel John
About this research
- This is an active NIH-funded research project — typically early-stage science, not a clinical trial accepting patient enrollment.
- Some NIH-funded labs run parallel clinical studies or seek volunteers for related work. To check, contact the principal investigator or institution listed above.
- For full project details, budget, and progress reports, visit the official NIH RePORTER page below.