Lentiviral-mediated genetic correction of hematopoietic and mesenchymal progenitor cells from Fanconi anemia patients.

@article{Jacome2009LentiviralmediatedGC,
  title={Lentiviral-mediated genetic correction of hematopoietic and mesenchymal progenitor cells from Fanconi anemia patients.},
  author={Ariana Jacome and Susana Navarro and Paula R{\'i}o and Rosa Ya{\~n}ez and Africa Gonz{\'a}lez-Murillo and Marta L Lozano and Mar{\'i}a Luisa Lamana and Juli{\'a}n Sevilla and Teresa Oliv{\'e} and Cristina D{\'i}az-Heredia and Isabel Badell and Jes{\'u}s M. Gonçalvez Estella and Lu{\'i}s Madero and Guillermo Guenechea and J{\'o}se Luis Casado and Jos{\'e} C Segovia and Juan Antonio Bueren},
  journal={Molecular therapy : the journal of the American Society of Gene Therapy},
  year={2009},
  volume={17 6},
  pages={1083-92}
}
Previous clinical trials based on the genetic correction of purified CD34(+) cells with gamma-retroviral vectors have demonstrated clinical efficacy in different monogenic diseases, including X-linked severe combined immunodeficiency, adenosine deaminase deficient severe combined immunodeficiency and chronic granulomatous disease. Similar protocols, however, failed to engraft Fanconi anemia (FA) patients with genetically corrected cells. In this study, we first aimed to correlate the… CONTINUE READING