A multicenter comparison of quantification methods for antisense oligonucleotide-induced DMD exon 51 skipping in Duchenne muscular dystrophy cell cultures.
Monika HillerMaria Sofia FalzaranoIker Garcia-JimenezValentina SardoneRuurd C VerheulLinda PopplewellKaren AnthonyEstibaliz Ruiz-Del-YerroHana OsmanJelle J GoemanKamel MamchaouiGeorge DicksonAlessandra FerliniFrancesco MuntoniAnnemieke Aartsma-RusVirginia Arechavala-GomezaNicole A DatsonPietro SpitaliPublished in: PloS one (2018)
Our results suggest that digital droplet PCR was the most precise and quantitative method. The quantification of exon 51 skipping by Agilent bioanalyzer after a single round of PCR was the second-best choice with a 2.3-fold overestimation of exon 51 skipping levels compared to digital droplet PCR.