Figure 2.
Comparison of exon 51 skipping in human wild-type myoblasts transduced by different lentiviral vectors. (a) Normal human myoblasts were transduced with lentiviral vectors encoding different U7 snRNA constructs and exon 51 skipping is assessed by nested reverse transcriptase–PCR. (b) Bands of the expected size were analyzed by sequencing, which confirmed the precise skipping of exon 51, joining exons 50 and 52. Note that additional bands between the nonskipped and skipped products are visible in some analyses. This is due to heteroduplex formation and has been described previously.34 AON, antisense oligonucleotide; AS, acceptor splice; DS, donor splice.