RNA data of human FOP patients-derived iPSCs treated with ACVR1 R206H mutant-targeting AAVs. RNA data of human FOP patients-derived iPSCs treated with ACVR1 R206H mutant-targeting AAVs

We found that both A9 (amiR-RH6) and A10 (amiR-RH7)-AAVs significantly decrease osteogenic differentiation of FOP-iPSCs. We also observed that A9 and A10-AAVs treatment changed the ratio of R206H mutant to wildtype of ACVR1 in FOP-iPSCs. To identify off-target effect of A9 or A10-AAVs, we compared RNA expression of FOP-iPSCs treated with control, A9, or A10-AAVs. Overall design: Comparative gene expression profiling analysis of RNA-seq data for human iPSCs treated with control, A9, or A10-AAVs.

我们发现，A9（amiR-RH6）与A10（amiR-RH7）型腺相关病毒（AAV，Adeno-associated virus）均能显著降低进行性骨化性纤维发育不良诱导多能干细胞（FOP-iPSCs，Fibrodysplasia Ossificans Progressiva-induced pluripotent stem cells）的成骨分化能力。
本研究同时观测到，经A9与A10-AAVs处理后，FOP-iPSCs中激活素A受体1型（ACVR1，Activin A Receptor Type 1）的R206H突变体与野生型的比例发生了改变。
为鉴定A9或A10-AAVs的脱靶效应，我们对经对照、A9或A10-AAVs处理的FOP-iPSCs开展了RNA表达水平的比较分析。
整体实验设计：对经对照、A9或A10-AAVs处理的人诱导多能干细胞的RNA测序（RNA-seq，RNA sequencing）数据开展比较性基因表达谱分析。