Hopping to a better protein. Science 5 Dec 2008, v. 322, p.1454-5

“Clinical trials are under way to test an innovative use of antisense technology to stem paralysis in Duchenne muscular dystrophy”

This article give progress updates, quotes from scientists involved in developing exon-skipping therapeutics, and describes how exon skipping works. They discuss the regulatory problems of having many different sequences needed to treat many different people each with their own unique mutation.

One complaint: they say that “Qi Long Lu, a pathologist at Carolinas Medical Center in Charlotte, North Carolina, has added a peptide rich in the amino acid arginine to the morpholino antisense.” Sorry, Science, you got that one wrong. That peptide was developed and added by my wife, Hong Moulton.