Exon 51 was at this stage in 2008. Hope it doesnt take this long for other exons.
“Systemic treatment of the majority of Duchenne dystrophy will require multiple sequences to be delivered in the blood, and this study also is the first proof-of-principle of multiple exon-skipping in any organism,” Shin’ichi Takeda, MD, another senior author, said. “In order to realize that promise in human trials, it also will be important to re-evaluate current measures of toxicity, efficacy, and marketing that ensure both safety for the patient, as well as rapid development and distribution of life-saving drugs.
I have a paper authored by Annemieke Aartsma-Rus that says 64% of all mutations are applicable to single exon skipping. 70% of deletions. Not sure why this paper says the majority would need multiple sequences. Any thoughts?
What they meant was that skipping more than one exon will benifit between 80 and 90 percent of patients.
Skipping one exon will still benifit up 64%, by skipping multiples it pushes it up to between 80-90%.