Author(s) : Cohen-Haguenauer, Odile , Péault, Bruno , Bauche, Cécile , Daniel, Marie-Thérèse , Casal, Ibrahim , Levy, Vincent , Dausset, Jean

Description : Fanconi anemia (FA) is a rare inherited genomic instability syndrome representing one of the best examples of hematopoietic stem cell deficiency. Although FA might be an excellent candidate for bone marrow (BM) genetic correction ex vivo, knockout animal models are not sufficient to guide preclinica...

Repository : Europe PubMed Central

Language(s) : English