Creating RNA treatments for rare brain disorders
Official title: Developing RNA therapeutics for rare neurodevelopmental disorders
Funded at Dayi Therapeutics, INC. · NIH-11239933 · Data updated 2026-11-12
This study is exploring new RNA-based treatments for rare brain disorders caused by single gene changes, aiming to help those affected by these conditions by restoring normal gene function and offering hope for better care.
Quick facts
| Grant type | NIH-funded research |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Dayi Therapeutics, INC. NIH-funded |
| Lab location | 1 site (College Station, United States) |
| Project start | May 1, 2023 |
| Project end | December 31, 2026 |
| Funding year | 2025 |
| Award (this year) | $853,987 |
| NIH project number | 4R42HD111138-02 |
| Project ID | NIH-11239933 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are individuals diagnosed with rare neurodevelopmental disorders linked to specific gene mutations.
✕Less likely to help
Patients with neurodevelopmental disorders that are not linked to single gene mutations may not benefit from this research.
What this research studies
This research focuses on developing RNA-based therapies to treat rare neurodevelopmental disorders caused by single gene mutations. These conditions, while individually rare, affect a significant portion of the population and often have no existing treatments. The approach involves using antisense oligonucleotides (ASOs) to modify gene expression and restore normal protein function. By targeting specific genes associated with severe developmental disabilities, the research aims to provide a new therapeutic option for affected patients.
Why it matters
Potential benefit: If successful, this research could lead to effective treatments for patients suffering from rare neurodevelopmental disorders, improving their quality of life.
How similar studies have gone: Other research has shown promise in using RNA-based therapies for genetic disorders, indicating potential success for this novel approach.
Where this research is happening
-
Dayi Therapeutics, INC.
College Station, United States Active
Researchers
- Principal investigator: Lotti, Francesco — Dayi Therapeutics, INC.
- Study coordinator: Lotti, Francesco
Related research
Other NIH-funded projects on the same topics.
-
Intergenerational factors in neurodevelopmental disorders
This project looks at how experiences before and during pregnancy across generations may influence the risk of neurodevelopmental conditions like ADHD, autism, and related disorders in grandchildren.
-
Understanding brain signals in autism to identify biologically distinct subtypes
This study uses EEG to link how the brain processes sensory information with behavior in young children with autism and related concerns.
-
Clinical Translational Research Center for Neurodevelopmental Disorders
A funded center supports research on neurodevelopmental disorders like Angelman syndrome and autism to better understand causes and improve treatments for children.
-
Synaptic nanostructure in neurodevelopmental disorders
This project aims to map the tiny architecture of brain synapses in native cells to understand how connections form and change in neurodevelopmental disorders.
-
Maternal acetaminophen use in pregnancy and ADHD risk in children
A large study will follow thousands of mother-child pairs to see if taking acetaminophen during pregnancy relates to ADHD in kids.
-
GDF11’s role in brain development and autism-related disorders
A new NIH grant tests whether adjusting a growth factor called GDF11 can improve brain function and behaviors in models related to autism and other neurodevelopmental disorders.
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.