Correcting hereditary tyrosinemia type 1 and other liver diseases using gene therapy.
Lifelong Correction of Hereditary Tyrosinemia Type 1 and Other Monogenic Liver Diseases using Liver-specific Hydrodynamic Gene Delivery through the Biliary System
This study is testing a new way to treat hereditary tyrosinemia type 1 (HT1) by using a special gene therapy that delivers healthy genes directly to the liver, which could help patients avoid liver transplants and long-term medications.
Quick facts
| Grant type | NIH-funded research |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Hydrogene Therapeutics INC NIH-funded |
| Lab location | 1 site (Houston, United States) |
| Project ID | NIH-11005449 on NIH RePORTER |
What this research studies
This research focuses on developing a new gene therapy approach to treat hereditary tyrosinemia type 1 (HT1) and other monogenic liver diseases. It utilizes a non-viral method to deliver functional genes directly to liver cells through the biliary system, aiming to replace the need for liver transplants and long-term medication. The therapy seeks to address the underlying genetic defect that causes HT1, potentially providing a lifelong solution for affected patients. By improving the delivery method, this research aims to enhance the safety and effectiveness of gene therapy for liver diseases.
Who could benefit from this research
Good fit: Ideal candidates for this research are individuals diagnosed with hereditary tyrosinemia type 1 or other similar monogenic liver diseases.
Not a fit: Patients with liver diseases not caused by genetic defects or those who do not have hereditary tyrosinemia type 1 may not benefit from this research.
Why it matters
Potential benefit: If successful, this research could provide a safe and effective lifelong treatment option for patients with hereditary tyrosinemia type 1, reducing the need for liver transplants and long-term medication.
How similar studies have performed: While gene therapy for liver diseases is an emerging field, this specific approach using hydrodynamic delivery via the biliary system is novel and has not been extensively tested in clinical settings.
Where this research is happening
Houston, United States
- Hydrogene Therapeutics INC — Houston, United States (Active)
Researchers
- Principal investigator: Kumbhari, Vivek — Hydrogene Therapeutics INC
- Study coordinator: Kumbhari, Vivek
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.