A prospective study in the rational design of efficient antisense oligonucleotides for exon skipping in the DMD gene.

ABSTRACT: Antisense oligonucleotide (AON)-mediated exon skipping to restore dystrophin expression in Duchenne muscular dystrophy (DMD) therapy shown promise in a number of human clinical trials. Current AON design methods are semi-empirical, involving either trial-and-error and/or preliminary experimentations. Therefore, a rational approach to design efficient AONs to address the wide spectrum of patients' mutations is desirable. Retrospective studies have extracted many AON design variables, but they were not tested prospectively to design AONs for skipping DMD exons. Not only did the variables differ among the various studies, no numerical cutoff for each variable was inferred, which makes their use in AON design difficult. The challenge is to thus select a minimal set of key independent variables

SUBMITTER: Pramono ZA 

PROVIDER: S-EPMC3404420 | biostudies-literature | 2012 Jul

REPOSITORIES: biostudies-literature