Clinical Research Directory
Browse clinical research sites, groups, and studies.
CRISPR-Edited HLA Donor Liver Transplant to Reduce Rejection
Sponsor: AMERICAN ORGAN TRANSPLANT AND CANCER RESEARCH INSTITUTE LLC
Summary
This early-phase clinical trial will assess the use of ex vivo CRISPR-Cas9 genome editing on donor liver grafts to reduce immunogenicity before transplantation. Donor livers will have HLA-A and HLA-B genes knocked out, and HLA class II expression disabled (by targeting the CIITA transactivator gene), aiming to create a "hypoimmunogenic" organ less prone to rejection. The edited liver is then transplanted into patients with end-stage liver disease. The primary focus is on safety and feasibility - determining whether a CRISPR-edited liver can be transplanted successfully and function normally - as well as evaluating reductions in immune response (acute rejection, anti-donor T cell activation) and graft function over time.
Official title: A Phase 1/2, Open-Label, Single-Arm Study to Evaluate the Safety, Immunogenicity Reduction, Transplant Function, and Feasibility of Ex Vivo CRISPR-Cas9 Gene-Edited Donor Liver Transplantation Targeting HLA Class I (HLA-A, HLA-B) and Class II (Via CIITA) Genes.
Key Details
Gender
All
Age Range
16 Years - 85 Years
Study Type
INTERVENTIONAL
Enrollment
90
Start Date
2025-06-01
Completion Date
2028-12-28
Last Updated
2025-07-08
Healthy Volunteers
No
Conditions
Interventions
Ex Vivo CRISPR-Cas9 Gene Editing of Donor Liver
Donor liver tissue is perfused outside the body with a CRISPR-Cas9 RNP complex targeting HLA-A, HLA-B, and CIITA, to create a hypoimmunogenic graft. After confirming successful gene knockout, the liver is transplanted into the patient following standard surgical techniques. Post-operative care includes routine immunosuppressive therapy with planned adjustments based on the patient's tolerance and evidence of graft immunogenicity.
Locations (1)
Peking University Health Science Center (PKUHSC)
Beijing, Changping, China