Table 1 Major inclusion and exclusion criteria for gene therapy in patients with biallelic FANCA germ-line mutations as proposed by International Fanconi Anemia Gene Therapy Working Group.

Inclusion Criteria

1. FA demonstrated by a positive test for increased sensitivity to chromosomal breakage with MMC/DEB and determination of FA complementation group A by somatic cell hybrids, molecular characterization, western blot analysis, direct FANCA sequencing, or acquisition of mitomycin C resistance after in vitro transduction with a vector bearing the FANCA cDNA.

2. Bone Marrow analysis demonstrating normal karyotype.

Exclusion Criteria

1. Uncontrolled infection (viral, bacterial, or fungal).

2. Patients with an HLA identical sibling donor.