Developing new RNA tools to target difficult diseases like cancer.
Modular Reagents for Programmable RNA Manipulation by Endogenous Proteins
['FUNDING_FELLOWSHIP'] · STANFORD UNIVERSITY · NIH-11012855
This study is exploring new RNA-based treatments that can specifically target and silence harmful RNA in diseases like cancer and genetic disorders, offering a gentler alternative to traditional gene editing methods.
Quick facts
| Phase | ['FUNDING_FELLOWSHIP'] |
|---|---|
| Study type | Nih_funding |
| Sex | All |
| Sponsor | STANFORD UNIVERSITY (nih funded) |
| Locations | 1 site (STANFORD, UNITED STATES) |
| Trial ID | NIH-11012855 on ClinicalTrials.gov |
What this research studies
This research focuses on creating innovative RNA-based molecules that can manipulate RNA in a targeted way, offering a new approach to treating challenging diseases such as cancers and genetic disorders. The project aims to develop bifunctional molecules that link RNA oligonucleotides to small molecules, allowing for programmable targeting of RNA without the permanent changes associated with gene editing. By utilizing a mechanism similar to small interfering RNA (siRNA), these molecules can bind to specific RNA targets and silence them, potentially leading to effective treatments. This approach seeks to overcome the limitations of current RNA manipulation tools that are difficult to deliver in the body.
Who could benefit from this research
Good fit: Ideal candidates for this research are patients with cancers, genetic disorders, or neurodegenerative diseases who may benefit from novel RNA-targeting therapies.
Not a fit: Patients with conditions that are not related to RNA manipulation or those who do not have the specific diseases targeted by this research may not receive any benefit.
Why it matters
Potential benefit: If successful, this research could lead to new, effective treatments for patients with hard-to-treat diseases like cancer and neurodegenerative disorders.
How similar studies have performed: Other research has shown success with RNA manipulation techniques, particularly with small interfering RNAs, indicating a promising avenue for therapeutic development.
Where this research is happening
STANFORD, UNITED STATES
- STANFORD UNIVERSITY — STANFORD, UNITED STATES (ACTIVE)
Researchers
- Principal investigator: LUSI, ROBERT FOLLETT — STANFORD UNIVERSITY
- Study coordinator: LUSI, ROBERT FOLLETT
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