A simplified approach to improve the efficiency and safety of ex vivo hematopoietic gene therapy in fanconi anemia patients.

@article{Jacome2006ASA,
  title={A simplified approach to improve the efficiency and safety of ex vivo hematopoietic gene therapy in fanconi anemia patients.},
  author={Ariana Jacome and Susana Navarro and Jose A Casado and Paula R{\'i}o and Lu{\'i}s Madero and Jes{\'u}s M. Gonçalvez Estella and Juli{\'a}n Sevilla and Isabel Badell and Juan J. Ortega and Teresa Oliv{\'e} and Helmut Hanenberg and Jos{\'e} C Segovia and Juan Antonio Bueren},
  journal={Human gene therapy},
  year={2006},
  volume={17 2},
  pages={245-50}
}
Fanconi anemia (FA) is an inherited DNA repair disorder characterized by genetic instability of cells lacking a functional FA/BRCA pathway. Previous studies have shown that in vitro stimulation of bone marrow cells (BMCs) from FA mice promotes apoptosis, reduces the reconstitution ability of the stem cells, and induces myelodysplasia and myeloid leukemia upon reinfusion of the cells. This suggests the convenience of adapting standard protocols of gene therapy to FA. Here we show that the… CONTINUE READING