hAPOC3

利用同源重组，将小鼠Apoc3基因进行人源化修饰。

Fig1. Detection of APOC3 expression in liver by RT-PCR. 

Wild type: only one band at 196 bp with primers F1/R1(mApoc3); 

Homozygous: only one band at 219 bp with primers F2/R2(hAPOC3). 

Abbr. M, DNA marker; HO, homozygous; WT, wild type.

Fig2. Detection of species-specific APOC3 expression in serum by ELISA. 

Abbr. HO, homozygous; HE, heterozygous; WT, wild type.

Fig3. Monitoring of blood lipids levels in hAPOC3 mice (n=3 female and 3 male). 

Abbr. Hom, homozygous; WT, wild type.

Fig4. In Vivo Efficacy of APOC3 RNAi (ARO-APOC3) in hAPOC3 Mice.

hAPOC3 mice (male, 13 weeks old) were randomly divided into two groups(n=5/group). Mice were administered with ARO-APOC3, a nucleic acid drug targeting hAPOC3, synthesized according to the relevant patents. Blood lipids level of hAPOC3 mice before or after dosing were detected. Compared to vehicle, ARO-APOC3 treatment group showed a decrease in TG, T-CHO and LDL-C. Mean ± SEM. t-test, *P < 0.05, ***P < 0.001.

Fig5. In Vivo Efficacy of APOC3 RNAi (ARO-APOC3) in hAPOC3 Mice.

hAPOC3 mice (male, 13 weeks old) were randomly divided into two groups(n=5/group). Mice were administered with ARO-APOC3, a nucleic acid drug targeting hAPOC3, synthesized according to the relevant patents. At day 29 post-dosing, the mice were euthanized, and their livers were collected for the assessment of human APOC3 mRNA expression via qPCR. The results indicated a significant reduction in human APOC3 expression in the ARO-APOC3 treatment group compared to the vehicle control. Mean ± SEM. t-test.

Fig6. In Vivo Efficacy of APOC3 RNAi (ARO-APOC3) in hAPOC3 Mice. 

hAPOC3 mice (male, 13 weeks old) were randomly divided into two groups(n=5/group). Mice were administered with ARO-APOC3, a nucleic acid drug targeting hAPOC3, synthesized according to the relevant patents. Expression level of hAPOC3 before or after dosing at indicated timepoint was detected by ELISA. Compared to vehicle, ARO-APOC3 treatment group showed a significant decrease in the expression of hAPOC3. Mean ± SEM. t-test, ***P < 0.001