Boosting PKD1 Gene Activity for Polycystic Kidney Disease
PKD1 derepression as a potential therapy for Polycystic Kidney Disease
['FUNDING_R01'] · UT SOUTHWESTERN MEDICAL CENTER · NIH-11120862
This work explores a new way to help people with Autosomal Dominant Polycystic Kidney Disease (ADPKD) by increasing the activity of a specific gene called PKD1.
Quick facts
| Phase | ['FUNDING_R01'] |
|---|---|
| Study type | Nih_funding |
| Sex | All |
| Sponsor | UT SOUTHWESTERN MEDICAL CENTER (nih funded) |
| Locations | 1 site (DALLAS, UNITED STATES) |
| Trial ID | NIH-11120862 on ClinicalTrials.gov |
What this research studies
We know that many people with ADPKD have lower levels of the PKD1 gene, which contributes to their kidney disease. Our goal is to find a new treatment that can raise these PKD1 levels, potentially slowing or stopping the disease. We believe that a specific molecule, miR-17, might be preventing the PKD1 gene from working properly. This project will test if blocking miR-17 can boost PKD1 activity and reduce cyst growth in models of ADPKD, paving the way for new therapies.
Who could benefit from this research
Good fit: This foundational work is for patients with Autosomal Dominant Polycystic Kidney Disease (ADPKD), particularly those whose disease is linked to reduced PKD1 gene activity.
Not a fit: Patients whose ADPKD is not related to PKD1 gene dosage or those with other forms of kidney disease may not directly benefit from this specific approach.
Why it matters
Potential benefit: If successful, this approach could lead to a new treatment that slows or stops the progression of ADPKD, potentially preventing kidney failure for many patients.
How similar studies have performed: Preliminary studies have shown promising results in improving PKD1 mRNA translation and reducing cyst growth in mouse and human ADPKD models, suggesting this is a novel yet supported direction.
Where this research is happening
DALLAS, UNITED STATES
- UT SOUTHWESTERN MEDICAL CENTER — DALLAS, UNITED STATES (ACTIVE)
Researchers
- Principal investigator: PATEL, VISHAL — UT SOUTHWESTERN MEDICAL CENTER
- Study coordinator: PATEL, VISHAL
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: Adult Polycystic Kidney Disease