1. Taniguchi, T. and D'Andrea, A. D., (2006). The molecular pathogenesis of fanconi anemia: recent progress. Blood. 107: 4223-33
  2. Casado, J. A., et al., (2007). A comprehensive strategy for the subtyping of Fanconi Anemia patients: conclusions from the Spanish Fanconi Anemia research network. J Med Genet. 44: 241-249
  3. Callen, E., et al., (2005). A common founder mutation in FANCA underlies the world's highest prevalence of Fanconi anemia in Gypsy families from Spain. Blood. 105: 1946-9
  4. Verlinsky, Y., Rechitsky, S., Schoolcraft, W., Strom, C. and Kuliev, A., (2001). Preimplantation diagnosis for Fanconi anemia combined with HLA matching. Jama. 285: 3130-3.
  5. Cavazzana-Calvo, M., et al., (2000). Gene therapy of human severe combined immunodeficiency (SCID)-X1 disease. Science. 288: 669-72.
  6. Aiuti, A., et al., (2002). Correction of ADA-SCID by stem cell gene therapy combined with. Science. 296: 2410-3.
  7. Gaspar, H. B., et al., (2004). Gene therapy of X-linked severe combined immunodeficiency by use of a pseudotyped gammaretroviral vector. Lancet. 364: 2181-7
  8. Ott, M. G., et al., (2006). Correction of X-linked chronic granulomatous disease by gene therapy, augmented by insertional activation of MDS1-EVI1, PRDM16 or SETBP1. Nat Med. 12: 401-9
  9. Hacein-Bey-Abina, S., et al., (2003). A serious adverse event after successful gene therapy for X-linked severe combined immunodeficiency. N Engl J Med. 348: 255-6
  10. Hacein-Bey-Abina, S., et al., (2003). LMO2-associated clonal T cell proliferation in two patients after gene therapy for SCID-X1. Science. 302: 415-9
  11. Waisfisz, Q., et al., (1999). Spontaneous functional correction of homozygous fanconi anaemia alleles reveals novel mechanistic basis for reverse mosaicism. Nat Genet. 22: 379-83.
  12. Gross, M., et al., (2002). Reverse mosaicism in Fanconi anemia: natural gene therapy via molecular self-correction. Cytogenet Genome Res. 98: 126-35
  13. Jacome, A., et al., (2006). A simplified approach to improve the efficiency and safety of ex vivo hematopoietic gene therapy in fanconi anemia patients. Hum Gene Ther. 17: 245-50