Faster lab pipelines to turn genetic test results into treatments
Preclinical/Co-Clinical Section
This project builds lab models and testing pipelines to clarify unclear genetic test results and speed new nucleic-acid treatments for people with rare genetic conditions.
Quick facts
| Grant type | NIH-funded research |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Baylor College of Medicine NIH-funded |
| Lab location | 1 site (Houston, United States) |
| Project ID | NIH-11248142 on NIH RePORTER |
What this research studies
If your genetic test comes back unclear, this project connects clinicians with lab scientists who use animals like fruit flies and mice to test whether a suspicious gene change causes disease. Researchers recreate patient variants in these models and study how the change affects cells and tissues. They also use that information to try targeted approaches such as antisense oligonucleotides and other nucleic-acid therapies in the lab before moving toward patient care. The goal is a faster, reliable pipeline for turning genomic findings into clearer diagnoses and candidate treatments.
Who could benefit from this research
Good fit: People with suspected or confirmed rare genetic disorders—especially those with variants of uncertain significance—are the ideal candidates to benefit from this work.
Not a fit: People without genetic conditions, individuals whose variants are already definitively classified, or those who cannot access collaborating centers are unlikely to benefit directly.
Why it matters
Potential benefit: Could help more people get clear genetic diagnoses and identify personalized treatment options, including antisense therapies.
How similar studies have performed: Model-organism research and antisense oligonucleotide therapies have led to successful treatments for some rare diseases, but scaling a rapid functional-testing-to-therapy pipeline is still an emerging effort.
Where this research is happening
Houston, United States
- Baylor College of Medicine — Houston, United States (Active)
Researchers
- Principal investigator: Burrage, Lindsay C — Baylor College of Medicine
- Study coordinator: Burrage, Lindsay C
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.