Improving protein levels in disorders caused by missing gene copies
Official title: Manipulating microRNA-target interactions to rescue protein levels in haploinsufficient disorders
Funded at Scripps Research Institute, the · NIH-11166202 · Data updated 2026-11-12
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 start | June 1, 2025 |
| Project end | May 31, 2028 |
| Funding year | 2025 |
| Award (this year) | $45,418 |
| NIH project number | 1F31NS143345-01 |
| Project ID | NIH-11166202 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are individuals diagnosed with neurodevelopmental disorders related to haploinsufficient genes.
✕Less likely to help
Patients with neurodevelopmental disorders not linked to haploinsufficient genes may not benefit from this research.
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.
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 gone: Preliminary tests have shown promise for similar approaches, indicating potential for success in this novel research.
Where this research is happening
-
Scripps Research Institute, the
La Jolla, United States Active
Researchers
- Principal investigator: Sievert, David — Scripps Research Institute, the
- Study coordinator: Sievert, David
Related research
Other NIH-funded projects on the same topics.
-
The Participant Center helps people join and stay in a nationwide health study
A nationwide program to support people in joining, contributing biosamples, and staying connected to a large health study in the United States.
-
Data Sharing and Bioinformatics Core for Viral Systems Biology
A center-led effort to manage, share, and interpret large viral systems biology data using open tools and a community-friendly portal.
-
Germline-targeted HIV vaccines using membrane liposome tech to trigger bnAbs
A new HIV vaccine approach aims to spark broadly neutralizing antibodies by showing MPER on membranes, tested in models and early studies to guide future human trials.
-
Antivirals for hemorrhagic fever viruses like LASV, SFTSV, and EBOV
A multi-institutional effort to find and validate antiviral compounds that block key viral proteins from LASV, SFTSV, and EBOV to help people at risk from serious hemorrhagic fevers.
-
Developing new antivirals for flaviviruses
A research team is working to create oral antiviral drugs targeting flaviviruses to treat infections that affect millions of people.
-
CoV inhibitors targeting non-enzymatic SARS-CoV-2 components
A multicenter research effort seeks antiviral ideas that block SARS-CoV-2 parts that don’t work through traditional enzymes, aiming to help people affected by COVID-19.
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.