Nongenotoxic conditioning for gene therapy and allogeneic transplantation in Fanconi anemia
Description: PROJECT NARRATIVE Genotoxicity and the development of leukemia has been a major concern with traditional chemo- or radiotherapy- based conditioning regimens; this risk is particularly high in patients with DNA-repair defects such as Fanconi anemia (FA). The development and use of nongenotoxic conditioning for these diseases would therefore be paradigm-shifting and have a dramatic impact for both gene therapy and allogeneic transplantation. Here we propose to develop such nongenotoxic conditioning regimens for gene therapy and allogeneic bone marrow transplantation in patients with FA and also develop a highly novel approach to eliminate any uncorrected or residual FA cells after gene therapy or transplantation.
Funding type: research
Funder scheme: R01
Start date: 2019-12-20
End date: 2025-11-30
Investigator: BRUCE R BLAZAR
Institution: FRED HUTCHINSON CANCER CENTER
Topic: DNA Repair Mechanisms
Subfield: Molecular Biology
Domain: Life Sciences
Funded outputs count: 100
Funder award ID: R01HL147324
OpenAlex ID: G4468582213