TY - JOUR AU - Fragall, Clayton T. AU - Adams, Abbie M. AU - Johnsen, Russell D. AU - Kole, Ryszard AU - Fletcher, Sue AU - Wilton, Steve D. PY - 2011 DA - 2011/10/20 TI - Mismatched single stranded antisense oligonucleotides can induce efficient dystrophin splice switching JO - BMC Medical Genetics SP - 141 VL - 12 IS - 1 AB - Antisense oligomer induced exon skipping aims to reduce the severity of Duchenne muscular dystrophy by redirecting splicing during pre-RNA processing such that the causative mutation is by-passed and a shorter but partially functional Becker muscular dystrophy-like dystrophin isoform is produced. Normal exons are generally targeted to restore the dystrophin reading frame however, an appreciable subset of dystrophin mutations are intra-exonic and therefore have the potential to compromise oligomer efficiency, necessitating personalised oligomer design for some patients. Although antisense oligomers are easily personalised, it remains unclear whether all patient polymorphisms within antisense oligomer target sequences will require the costly process of producing and validating patient specific compounds. SN - 1471-2350 UR - https://doi.org/10.1186/1471-2350-12-141 DO - 10.1186/1471-2350-12-141 ID - Fragall2011 ER -