Exploring proteins from viruses to improve gene therapy delivery
Interrogation of retroelement-derived proteins for functional gene transfer
['FUNDING_FELLOWSHIP'] · MASSACHUSETTS INSTITUTE OF TECHNOLOGY · NIH-10917287
This study is looking at how special proteins from viruses can help make gene therapy better for people with genetic diseases, especially those affecting the brain, by improving how the treatment gets delivered to the right cells without causing unwanted reactions.
Quick facts
| Phase | ['FUNDING_FELLOWSHIP'] |
|---|---|
| Study type | Nih_funding |
| Sex | All |
| Sponsor | MASSACHUSETTS INSTITUTE OF TECHNOLOGY (nih funded) |
| Locations | 1 site (CAMBRIDGE, UNITED STATES) |
| Trial ID | NIH-10917287 on ClinicalTrials.gov |
What this research studies
This research investigates how proteins derived from retroelements can be used to enhance the delivery of gene therapy treatments. By focusing on the development of non-immunogenic delivery vehicles, the project aims to improve the effectiveness of gene therapy for various genetic diseases, particularly neurological disorders. The approach involves studying how certain viral proteins can self-assemble and facilitate the targeted delivery of nucleic acids to cells. Patients may benefit from more effective treatments that can address the root causes of their genetic conditions.
Who could benefit from this research
Good fit: Ideal candidates for this research are individuals with genetic diseases, particularly those affecting the nervous system, who may benefit from advanced gene therapy techniques.
Not a fit: Patients with conditions that are not genetic in nature or those who do not respond to gene therapy may not receive benefits from this research.
Why it matters
Potential benefit: If successful, this research could lead to more effective gene therapies that can treat a wider range of genetic diseases with fewer side effects.
How similar studies have performed: Previous research has shown promise in using viral proteins for gene delivery, indicating that this approach may lead to significant advancements in the field.
Where this research is happening
CAMBRIDGE, UNITED STATES
- MASSACHUSETTS INSTITUTE OF TECHNOLOGY — CAMBRIDGE, UNITED STATES (ACTIVE)
Researchers
- Principal investigator: LASH, BLAKE — MASSACHUSETTS INSTITUTE OF TECHNOLOGY
- Study coordinator: LASH, BLAKE
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.