How cells keep glycogen from forming harmful clumps
Uncovering cellular mechanisms to keep glycogen water-soluble
['FUNDING_R01'] · UT SOUTHWESTERN MEDICAL CENTER · NIH-11330347
This research looks at whether changes to glycogen's chemistry and branching can keep it from forming insoluble clumps that harm people with certain rare glycogen-storage diseases.
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-11330347 on ClinicalTrials.gov |
What this research studies
If you or a family member has a rare glycogen storage disease, this work aims to find out why glycogen can clump into damaging polyglucosan bodies. Researchers use lab experiments and mouse models to study two features — phosphate attached to glycogen and how the glycogen molecules are branched — that seem to control whether glycogen stays dissolved. They will change these features genetically and in test tubes to see if that prevents clumping and restores normal glycogen function in tissues. That information could help scientists design future treatments to reduce organ damage from these deposits.
Who could benefit from this research
Good fit: People with rare glycogen-storage disorders that cause insoluble glycogen or polyglucosan body accumulation in liver, muscle, heart, or brain would be most relevant.
Not a fit: Patients with conditions unrelated to insoluble glycogen, or those looking for an immediate treatment, are unlikely to benefit directly since this is preclinical research.
Why it matters
Potential benefit: Could identify molecular targets to prevent or reduce glycogen clumping and ultimately lead to therapies that protect liver, muscle, heart, or brain function.
How similar studies have performed: Prior laboratory and mouse studies have linked glycogen phosphate and branching to solubility, but these findings have not yet been translated into proven human therapies.
Where this research is happening
DALLAS, UNITED STATES
- UT SOUTHWESTERN MEDICAL CENTER — DALLAS, UNITED STATES (ACTIVE)
Researchers
- Principal investigator: NITSCHKE, FELIX — UT SOUTHWESTERN MEDICAL CENTER
- Study coordinator: NITSCHKE, FELIX
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.