A multicenter comparison of quantification methods for antisense oligonucleotide-induced DMD exon 51 skipping in Duchenne muscular dystrophy cell cultures.

A multicenter comparison of quantification methods for antisense oligonucleotide-induced DMD exon 51 skipping in Duchenne muscular dystrophy cell cultures.

BACKGROUND Duchenne muscular dystrophy is a deadly illness brought on by lack of dystrophin. Skipping of exons adjoining to out-of-frame deletions has confirmed to revive dystrophin expression in Duchenne sufferers. Exon 51 has been essentially the most studied goal in each preclinical and medical settings and the provision of standardized procedures to quantify exon skipping …

A multicenter comparison of quantification methods for antisense oligonucleotide-induced DMD exon 51 skipping in Duchenne muscular dystrophy cell cultures. Read More »