Correction of Fanconi Anemia Group C Hematopoietic Stem Cells Following Intrafemoral Gene Transfer

Table 1

Number of mice that survived MMC-treatments after LV-mediated intrafemoral gene transfer.

	Wild type		𝐹𝑎𝑛𝑐𝐶^−/−		𝐹𝑎𝑛𝑐𝐴^−/−
		Pre-conditioned∗		Pre-conditioned∗		Preconditioned
	5/5	6/6	5/5	6/6	3/3	3/4{
Weekly MMC (0.3 mg /kg)†	5/5	6/6	5/5	5/6‡	0/3	0/4

Number of mice that survived/number of mice IF-injected with EGFP-FancC recombinant viral particles.
∗ Mice were pre-conditioned with MMC (0.3 mg/kg) 4 days prior to IF injection.
† Weekly treatment with MMC for 15 weeks.
‡ 1 𝐹𝑎𝑛𝑐𝐶^−/− mouse died due to bleeding during blood collection.
{ 1 𝐹𝑎𝑛𝑐𝐴^−/− mouse died 15 days following IF injection from a mishap unrelated to vector administration.