Using gene editing and lipid nanoparticles to treat hemophilia A
Gene Editing for Hemophilia A Treatment Using Lipid Nanoparticles
['FUNDING_R01'] · SEATTLE CHILDREN'S HOSPITAL · NIH-11163538
This study is testing a new way to treat hemophilia A by using tiny particles to deliver gene-editing tools that could fix the gene causing the bleeding disorder, aiming to offer a more lasting solution than the usual treatments.
Quick facts
| Phase | ['FUNDING_R01'] |
|---|---|
| Study type | Nih_funding |
| Sex | All |
| Sponsor | SEATTLE CHILDREN'S HOSPITAL (nih funded) |
| Locations | 1 site (SEATTLE, UNITED STATES) |
| Trial ID | NIH-11163538 on ClinicalTrials.gov |
What this research studies
This research aims to develop a new treatment for hemophilia A, a bleeding disorder caused by a deficiency in the factor VIII gene. By utilizing lipid nanoparticles to deliver CRISPR/Cas9 gene editing tools, the project seeks to correct the faulty gene responsible for this condition. Current treatments involve frequent and costly infusions of factor VIII protein, which are not always effective. This innovative approach could provide a more permanent solution by directly editing the gene in the patient's liver cells, potentially improving their quality of life.
Who could benefit from this research
Good fit: Ideal candidates for this research are individuals diagnosed with hemophilia A who have a deficiency in the factor VIII gene.
Not a fit: Patients with hemophilia B or other bleeding disorders unrelated to factor VIII may not benefit from this research.
Why it matters
Potential benefit: If successful, this research could lead to a long-lasting treatment for hemophilia A, reducing the need for frequent infusions and improving patient outcomes.
How similar studies have performed: Previous trials using adeno-associated viral gene therapy for hemophilia A have shown promising results, indicating that gene therapy approaches may be effective.
Where this research is happening
SEATTLE, UNITED STATES
- SEATTLE CHILDREN'S HOSPITAL — SEATTLE, UNITED STATES (ACTIVE)
Researchers
- Principal investigator: MIAO, CAROL H — SEATTLE CHILDREN'S HOSPITAL
- Study coordinator: MIAO, CAROL H
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: bleeding disorder