Finding new treatments for rare genetic disorders affecting glycosylation.
Identifying New Therapeutics and Molecular Mechanisms in Congenital Disorders of Glycosylation.
['FUNDING_OTHER'] · UNIVERSITY OF KANSAS LAWRENCE · NIH-11169410
This study is looking into rare genetic conditions called Congenital Disorders of Glycosylation (CDGs) to understand how they work and find new treatments, using human cells and tests on a small animal to see if any current medications can help improve health for people with these disorders.
Quick facts
| Phase | ['FUNDING_OTHER'] |
|---|---|
| Study type | Nih_funding |
| Sex | All |
| Sponsor | UNIVERSITY OF KANSAS LAWRENCE (nih funded) |
| Locations | 1 site (LAWRENCE, UNITED STATES) |
| Trial ID | NIH-11169410 on ClinicalTrials.gov |
What this research studies
This research focuses on Congenital Disorders of Glycosylation (CDGs), which are rare genetic conditions caused by errors in the glycosylation process, affecting how proteins are modified in the body. The study aims to uncover the mechanisms behind these disorders and identify potential new therapies. Researchers will use human cell cultures to explore how certain genes can be modified to improve health outcomes for patients with CDGs. Additionally, they will conduct drug screenings using a model organism to find existing medications that could be repurposed for treating these conditions.
Who could benefit from this research
Good fit: Ideal candidates for this research are individuals diagnosed with Congenital Disorders of Glycosylation, particularly those with DPAGT1-CDG.
Not a fit: Patients with other genetic disorders unrelated to glycosylation may not benefit from this research.
Why it matters
Potential benefit: If successful, this research could lead to new treatment options for patients suffering from Congenital Disorders of Glycosylation.
How similar studies have performed: While research on glycosylation disorders is ongoing, the specific approach of repurposing existing drugs for CDGs is relatively novel and has not been extensively tested.
Where this research is happening
LAWRENCE, UNITED STATES
- UNIVERSITY OF KANSAS LAWRENCE — LAWRENCE, UNITED STATES (ACTIVE)
Researchers
- Principal investigator: DALTON, HANS MARTIN — UNIVERSITY OF KANSAS LAWRENCE
- Study coordinator: DALTON, HANS MARTIN
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: Candidate Disease Gene