Targeting a gene linked to autism using innovative therapies
Official title: miRNA site-blocking ASOs as MeCP2 targeted therapeutics
Funded at Loyola University Chicago · NIH-10820414 · Data updated 2026-11-12
This study is exploring a new way to help people with autism-related disorders, like Rett syndrome, by using a special treatment that boosts a key gene in the brain, aiming for safer and more effective results.
Quick facts
| Grant type | R21 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Loyola University Chicago NIH-funded |
| Lab location | 1 site (Maywood, United States) |
| Project start | April 4, 2023 |
| Project end | March 31, 2026 |
| Funding year | 2024 |
| Award (this year) | $192,500 |
| NIH project number | 5R21HD111864-02 |
| Project ID | NIH-10820414 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research include individuals diagnosed with autism spectrum disorders, particularly those with Rett syndrome.
✕Less likely to help
Patients with autism who do not have mutations in the MeCP2 gene or those with unrelated neurodevelopmental disorders may not benefit from this research.
What this research studies
This research investigates a novel approach to treat autism-related disorders by targeting the Methyl-CpG Binding Protein 2 (MeCP2) gene, which is crucial for brain development. The study aims to enhance the expression of MeCP2 in a controlled manner using antisense oligonucleotides (ASOs) that block specific microRNAs, thereby fine-tuning protein levels to avoid adverse effects. By preventing the binding of repressive microRNAs to the gene's regulatory region, the researchers hope to achieve a therapeutic effect without the risks associated with traditional gene therapy methods. This approach could lead to safer and more effective treatments for conditions like Rett syndrome and other autism spectrum disorders.
Why it matters
Potential benefit: If successful, this research could provide a safer therapeutic option for patients with autism-related disorders by precisely regulating gene expression.
How similar studies have gone: While the approach of using ASOs to modulate gene expression is gaining traction, this specific application targeting MeCP2 is relatively novel and has not been extensively tested in clinical settings.
Where this research is happening
-
Loyola University Chicago
Maywood, United States Active
Researchers
- Principal investigator: Gogliotti, Rocco George — Loyola University Chicago
- Study coordinator: Gogliotti, Rocco George
Related research
Other NIH-funded projects on the same topics.
-
Identifying key cells and genes in children with MODS on ECMO
A high-tech study using single-cell analyses to find the important cells and genes that drive organ failure in critically ill children on ECMO support.
-
Preventing childhood B-ALL through maternal diet and TVA intake
A study at Loyola University Chicago will explore whether a mother’s intake of TVA during pregnancy can help prevent childhood B-ALL in babies.
-
Prenyltransferase inhibitor as a new therapy for inflammatory neuropathy
A new drug candidate called FGTI-2734 is being studied to calm nerve inflammation in conditions like AIDP/Guillain-Barré syndrome, with the goal of protecting nerves and reducing symptoms.
-
Leading causes of death in alcoholic ARDS patients using clinical data
This project uses big clinical data and AI to find the main factors that cause death in adults with ARDS who have alcohol use issues, aiming to improve understanding and care.
-
Affinity maturation in lung immune tissues in autoimmune disease
Aims to understand how immune cells in the lungs of people with autoimmune or allergic conditions refine their antibodies right where exposure happens.
-
Understanding how secreted messengers cause systemic inflammation in humans
A small NIH grant will study which secreted proteins drive systemic inflammation in living bodies, with a focus on conditions like sepsis and autoimmune diseases.
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.