Programmable tools to selectively edit disease-related proteins
Towards the Design of a Programmable, Isoform-Selective Proteome Editing System
['FUNDING_OTHER'] · UNIVERSITY OF PENNSYLVANIA · NIH-11390777
Researchers are building programmable protein-editing tools that could one day remove or stabilize harmful cancer-related proteins.
Quick facts
| Phase | ['FUNDING_OTHER'] |
|---|---|
| Study type | Nih_funding |
| Sex | All |
| Sponsor | UNIVERSITY OF PENNSYLVANIA (nih funded) |
| Locations | 1 site (PHILADELPHIA, UNITED STATES) |
| Trial ID | NIH-11390777 on ClinicalTrials.gov |
What this research studies
You should know this work is creating programmable protein editors that can find specific versions of proteins inside cells and either tag them for destruction or protect them. The team uses artificial intelligence to design short peptide guides and fuses those guides to enzymes that add or remove ubiquitin, giving an 'off' (degrade) or 'on' (stabilize) switch for target proteins. So far the experiments are being done in the lab using cells and molecular models at the University of Pennsylvania, not in people. Over time this approach aims to enable precise therapies for cancers driven by specific problematic proteins.
Who could benefit from this research
Good fit: Ideal future candidates would be patients with cancers driven by specific abnormal or mutant proteins who might enroll in later clinical trials testing these targeted proteome editors.
Not a fit: Patients needing immediate, proven treatments or those whose conditions are unrelated to protein dysfunction are unlikely to benefit from this early-stage laboratory research.
Why it matters
Potential benefit: If successful, this could lead to highly targeted ways to remove or restore disease-causing proteins, offering new treatment options for some cancers.
How similar studies have performed: CRISPR-based genome editing has shown clinical promise and early lab studies of peptide-guided protein degradation have worked in cells and models, but broad programmable proteome editing is still a novel and early-stage approach.
Where this research is happening
PHILADELPHIA, UNITED STATES
- UNIVERSITY OF PENNSYLVANIA — PHILADELPHIA, UNITED STATES (ACTIVE)
Researchers
- Principal investigator: CHATTERJEE, PRANAM — UNIVERSITY OF PENNSYLVANIA
- Study coordinator: CHATTERJEE, PRANAM
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: Cancers