Response to Buratti et al.: Can a ‘patch’ in a skipped exon make the pre-mRNA splicing machine run better?

In this issue, Buratti et al. discuss a recent paper on the possibility of using complementary sequences to target RS (arginine- and serine-rich) domains to defective splicing enhancers. They raise an important issue: does the method work in cells? We have developed a similar method and have demonstrated that it does indeed rescue splicing of exon 7 of the SMN2 gene in fibroblasts derived from a patient.