Finding new treatments for rare genetic disorders affecting glycosylation.
Official title: Identifying New Therapeutics and Molecular Mechanisms in Congenital Disorders of Glycosylation.
Funded at University of Kansas Lawrence · NIH-11169410 · Data updated 2026-11-12
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
| Grant type | NIH-funded research |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of Kansas Lawrence NIH-funded |
| Lab location | 1 site (Lawrence, United States) |
| Project start | September 1, 2024 |
| Project end | August 31, 2027 |
| Funding year | 2024 |
| Award (this year) | $249,000 |
| NIH project number | 4R00HD111662-03 |
| Project ID | NIH-11169410 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are individuals diagnosed with Congenital Disorders of Glycosylation, particularly those with DPAGT1-CDG.
✕Less likely to help
Patients with other genetic disorders unrelated to glycosylation may not benefit from this research.
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.
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 gone: 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
-
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.