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. Author manuscript; available in PMC: 2021 Feb 11.
Published in final edited form as: Ann Hematol. 2019 May 18;98(8):1905–1918. doi: 10.1007/s00277-019-03713-y

Figure 5. Pharmacokinetics of LNP-siRNA formulations suggests overall good stability and tolerability in mice.

Figure 5

A. Injection schedule for i.v injections of LNP-CTRL siRNA in normal NSG mice (red arrow indicates injection).

B. Percentage of Dil (LNP) positive cells as measured by flow cytometry in peripheral blood (PB) of NSG mice following treatment with PBS or LNP-CTRL siRNA at indicated time points. (mean ± SEM, n=3) (Red arrow indicates 5mg/kg LNP CTRL siRNA injection).

C. Mean fluorescent intensity of Dil (LNP) positive cells as measured by flow cytometry in peripheral blood (PB) of NSG mice following treatment with PBS or LNP-CTRL siRNA at indicated time points (mean ± SEM, n=3) (Red arrow indicates 5mg/kg LNP CTRL siRNA injection).

D. Mouse body weight following treatment (as indicated in Figure 5A) with PBS or LNP-CTRL siRNA at indicated time points. (mean ± SEM, n=3)

E. White blood cell count, (F) platelet count and (G) hemoglobin in peripheral blood of NSG mice treated with PBS or LNP-CTRL siRNA at indicated time points. (mean ± SEM, n=3).

H. Serum biochemical profile in LNP-CTRL siRNA and PBS treated control mice after 5 injections (serum was collected at 24 hours following the last injection) (K: potassium (mmol/l);Na: sodium (mmol/l); Cl: chloride (mmol/l);KREA: creatinine (μmol/l); UREA: urea (mmol/l); CK: creatine kinase (U/l);LDH: lactate dehydrogenase (U/l); (GLU: glucose (mmol/l);ALT: alanine aminotransferase (U/l); AST: aspartate aminotransferase (U/l);AMY: amylase (U/l);LIP: lipase (U/l);PRO: protein (g/l); ALB: albumin (g/l). (mean ± SEM, n=3 for PBS treated mice and n=6 for LNP-CTRL siRNA treated mice).

* indicates P<.05, ns, not significant.