Figure 3. SNALPs formulated miR-34a has anti-proliferative activity against MM in vitro and in vivo.

A) Trypan blue exclusion assay of SKMM-1 cells treated with SNALP-encapsulated miR-34a or scramble oligonucleotides as control (NC). Analysis was performed by microscope Burker chamber counts and trypan blue exclusion assay. Averaged values of three independent experiments are plotted including ±SD. P-values calculated by Student’s t test, two-tailed, at 24 and 48 hours after transfection, are respectively: 0.001 and 0.02 versus SNALP empty or 0.0099 and 0.01 versus SNALP miR-NC. B) Mice carrying palpable subcutaneous SKMM-1 tumor xenografts were treated by intravenous tail vein injections with 20 µg for each treatment of miR-34a encapsulated into SNALPs. As control SNALPs incapsulating scramble miR-NC or empty were used. Caliper measurement of tumors were taken every 2 days from the day of the enrollment. Averaged tumor volumes of 4 mice per group are reported±SD. (*) indicate significant P-values (P<0.05). D) Survival curves (Kaplan-Meier) of treated mice show prolongation of survival after SNALP formulated miR-34a treatment compared to controls (log-rank test, P = 0.0047 and 0.002 SNALP miR-34a vs empty and miR-NC, respectively). Survival was evaluated from the first day of treatment until death or sacrifice.