In vivo repopulation ability of genetically corrected bone marrow cells from Fanconi anemia patients.

ABSTRACT: 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 preclinical steps, and gene therapy attempts have proven disappointing so far. Contributing to these poor results is a characteristic and dramatic early BM-cells die-off when placed in culture. We show here that human primary FA BM cell survival can be ameliorated by using specific culture conditions that limit oxidative stress. When coupled with retrovirus-mediated transfer of the main complementation group FANCA-cDNA, we could achieve long-term reconstitution of the stem cell compartment both in vitro a

SUBMITTER: Cohen-Haguenauer O 

PROVIDER: S-EPMC1413721 | biostudies-literature | 2006 Feb

REPOSITORIES: biostudies-literature