Finding new medicines for TPI Deficiency
High-content screening for TPI Deficiency therapeutics
['FUNDING_R01'] · UNIVERSITY OF PITTSBURGH AT PITTSBURGH · NIH-11128708
This project looks for new medicines to help children with TPI Deficiency, a serious genetic condition that causes brain damage and other health problems.
Quick facts
| Phase | ['FUNDING_R01'] |
|---|---|
| Study type | Nih_funding |
| Sex | All |
| Sponsor | UNIVERSITY OF PITTSBURGH AT PITTSBURGH (nih funded) |
| Locations | 1 site (PITTSBURGH, UNITED STATES) |
| Trial ID | NIH-11128708 on ClinicalTrials.gov |
What this research studies
TPI Deficiency is a severe genetic condition affecting children, leading to problems like anemia, paralysis, and brain damage. This project aims to find new small molecule therapies by looking for compounds that can stabilize the TPI protein in affected cells. Researchers will use a special cell-based test to screen a large collection of potential medicines. Promising compounds will then be tested further in patient cells and animal models to see if they can effectively treat the disease.
Who could benefit from this research
Good fit: This foundational research is for patients with TPI Deficiency, particularly children, who currently lack effective treatment options.
Not a fit: Patients without TPI Deficiency would not directly benefit from this specific research.
Why it matters
Potential benefit: If successful, this work could lead to the first specific treatments for TPI Deficiency, potentially slowing or stopping the progression of this devastating disease.
How similar studies have performed: This approach uses a novel human cellular assay to screen for compounds, building on initial pilot screen success, and aims to discover entirely new therapies for TPI Deficiency.
Where this research is happening
PITTSBURGH, UNITED STATES
- UNIVERSITY OF PITTSBURGH AT PITTSBURGH — PITTSBURGH, UNITED STATES (ACTIVE)
Researchers
- Principal investigator: PALLADINO, MICHAEL JOHN — UNIVERSITY OF PITTSBURGH AT PITTSBURGH
- Study coordinator: PALLADINO, MICHAEL JOHN
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: Acquired brain injury