Gene therapy for sickle cell disease using a new viral approach
In vivo Sickle Cell Disease Gene Therapy Using a Novel Virus Like Particle
This study is testing a new gene therapy that uses a special delivery system to help people with sickle cell disease and β-thalassemia make healthy red blood cells, aiming to offer a long-lasting cure without the need for complicated hospital treatments.
Quick facts
| Grant type | Sbir 1 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Gigamune, INC. NIH-funded |
| Lab location | 1 site (San Francisco, United States) |
| Project ID | NIH-11004909 on NIH RePORTER |
What this research studies
This research focuses on developing a gene therapy for sickle cell disease (SCD) and β-thalassemia, which are genetic disorders affecting hemoglobin production. The approach involves using a novel virus-like particle to deliver genetic material directly into patients' cells, potentially allowing for the production of healthy red blood cells without the need for extensive hospital procedures. Unlike traditional methods that require the removal and editing of stem cells, this in vivo method can be administered in a more accessible manner at various hospitals. The goal is to provide a long-term cure for patients suffering from these conditions.
Who could benefit from this research
Good fit: Ideal candidates for this research are individuals aged 21 and older who have been diagnosed with sickle cell disease or β-thalassemia.
Not a fit: Patients with other forms of hemoglobinopathies or those who are not diagnosed with sickle cell disease or β-thalassemia may not benefit from this research.
Why it matters
Potential benefit: If successful, this research could lead to a life-long cure for patients with sickle cell disease and β-thalassemia.
How similar studies have performed: Other gene therapies for β-thalassemia have shown success, indicating that this novel approach may also be promising.
Where this research is happening
San Francisco, United States
- Gigamune, INC. — San Francisco, United States (Active)
Researchers
- Principal investigator: Johnson, David Scott — Gigamune, INC.
- Study coordinator: Johnson, David Scott
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.