LIUCHENG WU1,3,#, YI WANG2,#, LILI DU1, GUIQING JI1, RUI ZHOU2, ZEYI ZHAO2, JUN CHEN2, SHUNXING ZHU1,*
BIOCELL, Vol.45, No.6, pp. 1501-1507, 2021, DOI:10.32604/biocell.2021.09528
Abstract Spinal muscular atrophy (SMA) is an autosomal recessive hereditary neuromuscular disease. Exon 7 and 8 of survival of motor neuron 1 (SMN1) gene or only exon 7 homology deletion leads to the failure to produce a full-length SMN gene. The copy number of SMN2 gene with high homology of SMN1 affects the degree of disease and was the target gene for targeting therapy, in which splicing silencer in intron 7 was the key to suppress the inclusion of exon 7. In this study, we projected to use CRISPR/Case 9 for the targeted editing of intronic-splicing silencer (ISS) sequence to promote… More >
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