Improving protein levels in disorders caused by missing gene copies
Manipulating microRNA-target interactions to rescue protein levels in haploinsufficient disorders
This study is looking at ways to help people with neurodevelopmental disorders caused by missing a gene, by finding new methods to boost the production of important proteins that are usually suppressed, using cutting-edge tools like CRISPR.
Quick facts
| Grant type | Fellowship grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Scripps Research Institute, the NIH-funded |
| Lab location | 1 site (La Jolla, United States) |
| Project ID | NIH-11166202 on NIH RePORTER |
What this research studies
This research aims to address neurodevelopmental disorders caused by the loss of one gene copy, known as haploinsufficiency. It focuses on developing a new platform to identify and manipulate the interactions between microRNAs and these genes, which can suppress protein production. By using advanced techniques like CRISPR and single-cell analysis, the researchers will explore how to block these suppressive interactions to restore normal protein levels. This innovative approach could lead to significant advancements in treating conditions linked to haploinsufficient genes.
Who could benefit from this research
Good fit: Ideal candidates for this research are individuals diagnosed with neurodevelopmental disorders related to haploinsufficient genes.
Not a fit: Patients with neurodevelopmental disorders not linked to haploinsufficient genes may not benefit from this research.
Why it matters
Potential benefit: If successful, this research could lead to new therapies that restore normal protein levels in patients with neurodevelopmental disorders.
How similar studies have performed: Preliminary tests have shown promise for similar approaches, indicating potential for success in this novel research.
Where this research is happening
La Jolla, United States
- Scripps Research Institute, the — La Jolla, United States (Active)
Researchers
- Principal investigator: Sievert, David — Scripps Research Institute, the
- Study coordinator: Sievert, David
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.