Genetic & Genomic MedicinemedRxivPreprint
Multi-lineage natural gene therapy mediated by embryonic triploid mosaicism in the context of Fanconi anaemia
Michael F. Sharp, Caitlin Harris, Chirantani Mukherjee…·Wayne Crismani
A patient with Fanconi anemia developed triploid hematopoietic stem cells that lacked the disease phenotype, enabling sustained hematopoiesis through restored DNA repair.
DOI 10.1101/2025.10.29.25337140