NIH-funded research R01 grant

Exosome delivery of CRISPR/Cas9 to target hepatitis B in liver cells

Official title: Engineered exosomes carrying synthetic gRNA/Cas9 targeting HBV-infected cells

Funded at East Tennessee State University · NIH-11289436 · Data updated 2026-11-12

Testing a non-viral exosome delivery of CRISPR/Cas9 to remove hepatitis B virus DNA from infected liver cells.

Quick facts

Grant typeR01 grant
Study typeNIH-funded research
Funding institutionEast Tennessee State University
NIH-funded
Lab location1 site (Johnson City, United States)
Project startApril 15, 2024
Project endFebruary 28, 2029
Funding year2026
Award (this year)$497,714
NIH project number5R01AI177624-03
Project IDNIH-11289436 on NIH RePORTER

Who could benefit from this research

Could help

People with chronic hepatitis B infection, especially those with persistent viral DNA despite current antiviral therapy, would be the likely candidates for future trials.

Less likely to help

People without active HBV infection (for example, vaccinated or previously resolved infections) or those with irreversible end-stage liver failure are unlikely to benefit from this approach.

What this research studies

Researchers are developing tiny natural particles called exosomes to carry CRISPR/Cas9 gene-editing tools into HBV-infected liver cells. They will design synthetic guide RNAs and load Cas9 protein into exosomes, then test targeting, antiviral activity, biodistribution, and safety in cell and animal models. The team aims to avoid viral delivery methods to reduce insertional risks and off-target effects while improving delivery to hepatocytes. If the approach shows good specificity and safety in preclinical tests, it could set the stage for future human trials.

Why it matters

Potential benefit: If successful, this approach could clear the viral DNA that keeps hepatitis B alive and move toward a one-time curative therapy.

How similar studies have gone: CRISPR-based antiviral approaches have shown promise in cells and animal models, but using engineered exosomes to deliver gRNA/Cas9 is relatively new and less tested.

Where this research is happening

  • East Tennessee State University
    Johnson City, United States
    Active

Researchers

  • Principal investigator: Yao, Zhi Q. — East Tennessee State University
  • Study coordinator: Yao, Zhi Q.

About this research

  1. This is an active NIH-funded research project — typically early-stage science, not a clinical trial accepting patient enrollment.
  2. Some NIH-funded labs run parallel clinical studies or seek volunteers for related work. To check, contact the principal investigator or institution listed above.
  3. For full project details, budget, and progress reports, visit the official NIH RePORTER page.
Last reviewed 2026-11-14 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.