Creating new synthetic nucleic acids to treat sickle cell disease
Developing next generation synthetic nucleic acid analogous for sickle cell disease gene editing
['FUNDING_R01'] · UNIVERSITY OF CONNECTICUT STORRS · NIH-10747883
This study is exploring a new way to use special synthetic molecules to help fix the genetic issues that cause sickle cell disease, and it's designed for people with this condition who are looking for safer treatment options.
Quick facts
| Phase | ['FUNDING_R01'] |
|---|---|
| Study type | Nih_funding |
| Sex | All |
| Sponsor | UNIVERSITY OF CONNECTICUT STORRS (nih funded) |
| Locations | 1 site (STORRS-MANSFIELD, UNITED STATES) |
| Trial ID | NIH-10747883 on ClinicalTrials.gov |
What this research studies
This research focuses on developing innovative synthetic nucleic acids that can be used for gene editing to treat sickle cell disease. The approach involves using chemically modified triplex-forming peptide nucleic acids and donor DNAs, which are delivered intravenously through nanoparticles. This method aims to correct the genetic mutations responsible for sickle cell disease while minimizing the risk of harmful off-target effects that can occur with traditional gene editing techniques. The research will be conducted in a controlled environment to assess the effectiveness and safety of this new treatment strategy.
Who could benefit from this research
Good fit: Ideal candidates for this research are adults aged 21 and older who have been diagnosed with sickle cell disease.
Not a fit: Patients with other forms of anemia or genetic disorders unrelated to sickle cell disease may not benefit from this research.
Why it matters
Potential benefit: If successful, this research could provide a safer and more effective gene editing treatment for patients with sickle cell disease.
How similar studies have performed: Previous research has shown promising results using similar synthetic nucleic acid approaches in animal models, indicating potential for success in human applications.
Where this research is happening
STORRS-MANSFIELD, UNITED STATES
- UNIVERSITY OF CONNECTICUT STORRS — STORRS-MANSFIELD, UNITED STATES (ACTIVE)
Researchers
- Principal investigator: BAHAL, RAMAN — UNIVERSITY OF CONNECTICUT STORRS
- Study coordinator: BAHAL, RAMAN
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: Hb SS disease, HbSS disease, Hemoglobin S Disease, Hemoglobin sickle cell disease, Hemoglobin sickle cell disorder