A hallmark of Fanconi anemia is accelerated decline in hematopoietic stem and progenitor cells (CD34 +) leading to bone marrow failure. patients in 2011 (cDNA regulated by a human phosphoglycerate kinase (hPGK) promoter; ii) a short, overnight transduction to minimize cDNA (pRSC-PGK.FANCA-sW), both regulated by an hPGK promoter. Research-grade vectors were produced by the Fred… Continue reading A hallmark of Fanconi anemia is accelerated decline in hematopoietic stem