Table 1.

Effect of Small Interfering RNA Specifically Targeting PML-RARα on Development of Disease after Intraperitoneal Injection of NB4 Cells^a

	Cells transplanted
	No siRNA	siPML-RARα/S	siPML-RARα/L
Mice with disease/mice per group	3/3	3/4	0/5

NOD/SCID, nonobese diabetic/severe combined immunodeficient; siRNA, small interfering RNA; siPML-RARα/L, siRNA targeting the promyelocytic leukemia–retinoic acid α receptor fusion protein (long isoform); siPML-RARα/S, siRNA targeting PML-RARα (short isoform).

^aNB4 cells (3×10⁵) transduced with lentiviral vectors delivering siRNA as described in Fig. 2 or left untransduced were injected into NOD/SCID mice by the intraperitoneal route. Animals in the control group that received cells not expressing siRNA were analyzed on death 73–76 days postinjection; remaining mice were killed 180 days postinjection, at which time two of the four animals in the siPML-RARα/S group had developed signs of disease on visual inspection and three of four animals displayed disease on necropsy. No animals in the siPML-RARα/L group had developed signs of disease on either visual inspection or on necropsy.