Gene therapy to increase fetal hemoglobin and decrease sickle hemoglobin in sickle cell disease patients
Gene therapy targeting BCL11A to induce fetal hemoglobin and reduce sickle hemoglobin in patients with Sickle Cell Disease
This study is looking at a new gene therapy that helps people with sickle cell disease by boosting a type of hemoglobin that can make them feel better and reduce complications from their condition.
Quick facts
| Grant type | NIH-funded research |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Boston Children's Hospital NIH-funded |
| Lab location | 1 site (Boston, United States) |
| Project ID | NIH-10083551 on NIH RePORTER |
What this research studies
This research investigates a gene therapy approach that targets the BCL11A gene to enhance the production of fetal hemoglobin in patients with sickle cell disease. By increasing fetal hemoglobin levels, the therapy aims to reduce the harmful effects of sickle hemoglobin, which causes various complications in affected individuals. Patients will be monitored for changes in hemoglobin levels and overall health outcomes as part of the treatment process. The methodology involves advanced genetic techniques to modify the expression of specific genes related to hemoglobin production.
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 who do not have sickle cell disease or are under the age of 21 may not receive any benefit from this research.
Why it matters
Potential benefit: If successful, this research could significantly improve the quality of life for patients with sickle cell disease by reducing symptoms and complications associated with the condition.
How similar studies have performed: Previous research has shown promising results with gene therapy approaches targeting hemoglobin production, indicating potential for success in this area.
Where this research is happening
Boston, United States
- Boston Children's Hospital — Boston, United States (Active)
Researchers
- Principal investigator: Williams, David a — Boston Children's Hospital
- Study coordinator: Williams, David a
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.