From “journalism” to science you can understand.
In case you haven’t picked up on the “breaking news” of a “DNA ‘Patch’ for Canine Form of Muscular Dystrophy,” allow me to be the one to burst their bubble.
1) The “technology” they call “exon skipping” isn’t really that new, this clinical usage is new, but it’s been used before, mainly to produce gene knockdowns. [edit: knockout mutants require DNA to be modified]
2) They aren’t called “molecular ‘patches,’” they are called morpholinos (which they do call them much later), which are nitrogenous bases bound to a morpholine ring which prevents RNase H from degrading it. Other types of oligos have been used to produce knockdowns and knockout mutants for decades, but done via electroporation rather than transmembranous peptides.
3) In order to get these morpholinos to have any effect, they must be bound to a peptide capable of crossing the cell membrane; (or as Jon pointed out, high doses can also work) once in the cytoplasm, this peptide must release the morpholino and it will diffuse into the nucleus.
4) Because the mutations associated with muscular dystrophy are still being explored, not all types can be tested for at this time; we need to know what segment of the RNA sequence needs to be modified before being able to modify them.
In short, the “new technology” is not the morpholino itself, but the method of getting bloodstream morpholinos into the cell nucleus. We’ve been able to “patch” DNA for decades, doing so without electroporation or micro-injection with low doses is the key.