Multi-lineage natural gene therapy mediated by embryonic triploid mosaicism in the context of Fanconi anaemia
Michael F. Sharp, Caitlin Harris, Chirantani Mukherjee, Stevan Novakovic, Elissah Granger, Roser M. Pujol, Gerard Muñoz‐Pujol, Elva Z. Shi, Karen A. Dun, Cesar Salinas‐La Rosa, Zhisheng Xu, Mark Domenic Pertile, Krystle Standen, Rebecca Walsh, Andrew J. Deans, , Wayne Crismani
A patient with Fanconi anemia developed triploid hematopoietic stem cells that lacked the disease phenotype, enabling sustained hematopoiesis through restored DNA repair.
medRxiv · Oct 29, 2025 · PreprintGenetic & Genomic Medicine