Improving Gene Therapy for Hemophilia A
Defining the therapeutic efficacy, tolerogenic potential, and genotoxicity of liver-targeted AAV gene therapy for hemophilia A
This project aims to make gene therapy safer and more effective for people with hemophilia A, potentially even before birth.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Wake Forest University Health Sciences NIH-funded |
| Lab location | 1 site (Winston-Salem, United States) |
| Project ID | NIH-11169708 on NIH RePORTER |
What this research studies
Gene therapy offers the hope of a lasting solution for hemophilia A with a single treatment. However, current gene therapies for adults have shown some risks, like liver issues and potential genetic changes, and many patients already have immunity to the treatment delivery method. This work explores new ways to deliver gene therapy, including during pregnancy, to overcome these challenges and prevent the body from rejecting the treatment or developing new problems. The goal is to find a way to safely correct hemophilia A early in life, potentially avoiding complications seen in adult treatments.
Who could benefit from this research
Good fit: This foundational research does not directly recruit patients, but future clinical applications would target individuals with hemophilia A, including those with pre-existing immunity to AAV vectors or those who might benefit from early intervention.
Not a fit: Patients who do not have hemophilia A or related blood coagulation disorders would not directly benefit from this specific gene therapy approach.
Why it matters
Potential benefit: If successful, this work could lead to a safer, more effective, and potentially lifelong gene therapy for hemophilia A, possibly even for newborns or before birth.
How similar studies have performed: While gene therapy for hemophilia A has shown promise in clinical trials, this project addresses known challenges like immune responses and potential side effects to improve upon existing approaches.
Where this research is happening
Winston-Salem, United States
- Wake Forest University Health Sciences — Winston-Salem, United States (Active)
Researchers
- Principal investigator: Almeida-Porada, Graca Duarte — Wake Forest University Health Sciences
- Study coordinator: Almeida-Porada, Graca Duarte
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.