Antisense mediated exon skipping therapy for duchenne muscular dystrophy (DMD)

TY - JOUR

T1 - Antisense mediated exon skipping therapy for duchenne muscular dystrophy (DMD)

AU - Brolin, Camilla

AU - Shiraishi, Takehiko

PY - 2011/1/1

Y1 - 2011/1/1

N2 - Duchenne Muscular Dystrophy (DMD) is a lethal disease caused by mutations in the dystrophin gene (DMD) that result in the absence of essential muscle protein dystrophin. Among many different approaches for DMD treatment, exon skipping, mediated by antisense oligonucleotides, is one of the most promising methods for restoration of dystrophin expression. This approach has been tested extensively targeting different exons in numerous models both in vitro and in vivo. During the past 10 years, there has been a considerable progress by using DMD animal models involving three types of antisense oligonucleotides (2'-O-methyl phosphorothioate (2OME-PS), phosphorodiamidate morpholino oligomer (PMO)) and peptide nucleic acid (PNA).

AB - Duchenne Muscular Dystrophy (DMD) is a lethal disease caused by mutations in the dystrophin gene (DMD) that result in the absence of essential muscle protein dystrophin. Among many different approaches for DMD treatment, exon skipping, mediated by antisense oligonucleotides, is one of the most promising methods for restoration of dystrophin expression. This approach has been tested extensively targeting different exons in numerous models both in vitro and in vivo. During the past 10 years, there has been a considerable progress by using DMD animal models involving three types of antisense oligonucleotides (2'-O-methyl phosphorothioate (2OME-PS), phosphorodiamidate morpholino oligomer (PMO)) and peptide nucleic acid (PNA).

U2 - 10.4161/adna.2.1.15425

DO - 10.4161/adna.2.1.15425

M3 - Journal article

C2 - 21686247

SN - 1949-095X

VL - 2

SP - 6

EP - 15

JO - Artificial DNA: PNA and XNA

JF - Artificial DNA: PNA and XNA

IS - 1

ER -