NDUFS2 knockout human cell line for Complex I research
This is a genetically engineered human cell line in which the NDUFS2 gene has been permanently switched off, preventing the cell from producing a key protein component of mitochondrial Complex I, the main energy-generating machinery in human cells. Because Complex I dysfunction is linked to serious inherited metabolic diseases, researchers need a standardized human cell model where Complex I is reliably broken in a specific way. This cell line provides exactly that: a controlled experimental platform for studying how Complex I assembles and what goes wrong when NDUFS2 is missing. It can also serve as a screening tool to test whether candidate drugs or gene therapies can restore or compensate for lost Complex I activity.
What you could build
A licensed research cell line sold or sublicensed to pharmaceutical and biotech companies running drug-discovery programs targeting mitochondrial Complex I disorders; academic core facilities and CROs focused on rare metabolic disease would also be direct buyers.
Who in Virginia should care
Biotech and rare-disease drug developers in the Northern Virginia/DC corridor, as well as academic medical centers like UVA and VCU with mitochondrial disease research programs, would be natural licensees or collaborators.
Readiness: Lab validated
Concept — described but not yet demonstrated. Lab validated — supported by experimental results in the patent. Prototype likely — the text describes a built, working embodiment.
Readiness is inferred from the patent text, not from a lab visit.
The record
- Inventors
- Aloka B. BANDARA, David A. BROWN
- Filed
- Patent pending — filed February 1, 2022
- Status
- Application
- Publication number
- US20220299500A1
Ready to talk?
Virginia Tech Intellectual Properties handles licensing for this technology. Email us for an introduction prosim@harbor.capital.
Prosim summaries are generated from public patent text and are not legal advice.