Establishment of spinal muscular atrophy cell model by RNAi / 中南大学学报(医学版)

ABSTRACT

OBJECTIVE@#To establish spinal muscular atrophy (SMA) cell model by blocking the expression of SMN1 gene with shRNA.@*METHODS@#The recombinant SMN1 shRNA expression vector was constructed. SMA cell model was established by human mesenchymal stem cells(hMSCs) that the vector was transfected into were differentiated to neuron like cells (NLCs).At the same time the control groups were established that the shRNA-0 vector was transfected into and no vector was transfected into. The expression of fl-SMN and delta7-SMN mRNA was observed by RT-PCR analysis. The expression of fl-SMN protein was detected by Western blot.@*RESULTS@#The cells of all the groups were neuron like cells after being differentiated and the protein expression of NSE and NF was positive. The expression of fl-SMN and delta7-SMN mRNA and protein of NLCs in each group was upregulated (P0.05).@*CONCLUSION@#The NLCs, which recombinant SMN1 shRNA expression vector was transfected into, can be regarded as SMA cell model.