Using exosomes to improve gene therapy for heart diseases
AAV-Exosomes: Escaping Neutralizing Antibody and Enhancing Delivery
This study is exploring a new way to improve gene therapy for heart problems by using tiny bubbles called exosomes to safely deliver helpful genes to heart cells, which could lead to better treatments for people with inherited heart diseases and heart failure.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Icahn School of Medicine at Mount Sinai NIH-funded |
| Lab location | 1 site (New York, United States) |
| Project ID | NIH-10428364 on NIH RePORTER |
What this research studies
This research focuses on enhancing gene therapy for heart conditions by using adeno-associated vectors (AAV) encapsulated in exosomes. These exosomes help protect the AAV from neutralizing antibodies that can hinder treatment effectiveness. By investigating this method, the research aims to improve the delivery of therapeutic genes directly to heart cells, potentially leading to better outcomes for patients with inherited heart diseases and heart failure. The approach combines advanced techniques in gene therapy with innovative delivery systems to overcome existing challenges.
Who could benefit from this research
Good fit: Ideal candidates for this research are individuals with inherited cardiomyopathies or other types of heart failure who may benefit from advanced gene therapy.
Not a fit: Patients with heart conditions not related to genetic factors or those who do not have pre-existing antibodies may not receive benefit from this research.
Why it matters
Potential benefit: If successful, this research could lead to more effective gene therapies for patients suffering from heart diseases, improving their treatment options and outcomes.
How similar studies have performed: Other research has shown promise in using exosomes for gene delivery, indicating that this approach could be a significant advancement in the field.
Where this research is happening
New York, United States
- Icahn School of Medicine at Mount Sinai — New York, United States (Active)
Researchers
- Principal investigator: Sahoo, Susmita — Icahn School of Medicine at Mount Sinai
- Study coordinator: Sahoo, Susmita
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.