Reelin protein in Fragile X syndrome
Reelin and Fragile X Syndrome
['FUNDING_R21'] · UNIVERSITY OF SOUTH FLORIDA · NIH-11253286
The team is testing whether boosting a brain protein called Reelin can improve brain connections and help people with Fragile X syndrome.
Quick facts
| Phase | ['FUNDING_R21'] |
|---|---|
| Study type | Nih_funding |
| Sex | All |
| Sponsor | UNIVERSITY OF SOUTH FLORIDA (nih funded) |
| Locations | 1 site (TAMPA, UNITED STATES) |
| Trial ID | NIH-11253286 on ClinicalTrials.gov |
What this research studies
If you or a loved one has Fragile X syndrome, this project looks at whether adding or increasing Reelin can fix the disrupted brain signaling that underlies learning, anxiety, and hyperactivity. Researchers will use viral vectors and laboratory models to deliver Reelin and then measure changes in brain synapses and behavior that relate to cognition and social interactions. The effort follows earlier gene-therapy work that restored missing FMRP but often did not correct behavior, so this approach targets synaptic function as an alternative path toward symptom improvement. Results would guide whether Reelin-based therapies should move toward human testing.
Who could benefit from this research
Good fit: People with a confirmed diagnosis of Fragile X syndrome (FMR1 mutation) or caregivers of such individuals would be the primary group most likely considered for related future clinical efforts.
Not a fit: Individuals without Fragile X syndrome or whose symptoms are due to other genetic or acquired conditions are unlikely to benefit from this specific approach.
Why it matters
Potential benefit: If successful, this work could lead to a new approach that improves learning, social behavior, and other core symptoms in people with Fragile X syndrome.
How similar studies have performed: Previous gene therapies that restored FMRP often failed to rescue behavior, and Reelin-based strategies are relatively new with encouraging preclinical signs but no proven benefit in humans yet.
Where this research is happening
TAMPA, UNITED STATES
- UNIVERSITY OF SOUTH FLORIDA — TAMPA, UNITED STATES (ACTIVE)
Researchers
- Principal investigator: NASH, KEVIN RON — UNIVERSITY OF SOUTH FLORIDA
- Study coordinator: NASH, KEVIN RON
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.
Conditions: Alzheimer disease dementia, Alzheimer syndrome, Alzheimer's Disease