New Ways to Find Medicines and Fix Genes for Diseases
Integrating Chemistry and Evolution to Illuminate Biology and Enable Novel Therapeutics
This project explores new chemical methods and gene editing tools to develop treatments for human genetic diseases like Aran-Duchenne disease.
Quick facts
| Grant type | NIH-funded research |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Broad Institute, INC. NIH-funded |
| Lab location | 1 site (Cambridge, United States) |
| Project ID | NIH-11070341 on NIH RePORTER |
What this research studies
Our team is working on innovative ways to create new medicines and correct genetic errors. We use a method called DNA-templated synthesis to build and find small molecules that can block harmful proteins in the body. We also developed advanced gene editing tools, prime editing and base editing, which can precisely fix genetic mutations without causing major damage to DNA. These technologies are being applied to improve animal models of human genetic diseases, aiming to find new small-molecule drugs and gene-editing therapies.
Who could benefit from this research
Good fit: This foundational research is not directly recruiting patients but focuses on understanding and treating human genetic diseases.
Not a fit: Patients seeking immediate treatment options may not directly benefit from this early-stage research.
Why it matters
Potential benefit: If successful, this work could lead to entirely new medications and gene therapies for patients with genetic conditions, offering hope for diseases currently without effective treatments.
How similar studies have performed: The researchers have already successfully developed DNA-templated synthesis and advanced genome editing technologies, and discovered novel inhibitors in prior work.
Where this research is happening
Cambridge, United States
- Broad Institute, INC. — Cambridge, United States (Active)
Researchers
- Principal investigator: Liu, David R — Broad Institute, INC.
- Study coordinator: Liu, David R
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.