Functional correction in mouse models of muscular dystrophy using exon-skipping tricyclo-DNA oligomers

Dose-Dependent Pharmacokinetic Profiles of 2′-O-Methyl Phosphorothioate Antisense Oligonucleotidesin mdx Mice

(2015) Ingrid E.C. Verhaart et al.   Nucleic Acid Therapeutics

What Can We Learn From Clinical Trials of Exon Skipping for DMD?

(2014) Qi-long Lu et al.   Molecular Therapy-Nucleic Acids

Eteplirsen for the treatment of Duchenne muscular dystrophy

(2013) Jerry R. Mendell et al.   ANNALS OF NEUROLOGY

TricycloDNA-modified oligo-2′-deoxyribonucleotides reduce scavenger receptor B1 mRNA in hepatic and extra-hepatic tissues—a comparative study of oligonucleotide length, design and chemistry

(2012) Sue Murray et al.   NUCLEIC ACIDS RESEARCH

Exon skipping and dystrophin restoration in patients with Duchenne muscular dystrophy after systemic phosphorodiamidate morpholino oligomer treatment: an open-label, phase 2, dose-escalation study

(2011) Sebahattin Cirak et al.   LANCET

Systemic Administration of PRO051 in Duchenne's Muscular Dystrophy

(2011) Nathalie M. Goemans et al.   NEW ENGLAND JOURNAL OF MEDICINE

Chronic Systemic Therapy With Low-dose Morpholino Oligomers Ameliorates the Pathology and Normalizes Locomotor Behavior in mdx Mice

(2010) Alberto Malerba et al.   MOLECULAR THERAPY

Reduction of abnormal behavioral response to brief restraint by information from other mice in dystrophin-deficient mdx mice

(2010) Kazuhiro Yamamoto et al.   NEUROMUSCULAR DISORDERS

Position-dependent effects on stability in tricyclo-DNA modified oligonucleotide duplexes

(2010) D. Ittig et al.   NUCLEIC ACIDS RESEARCH

In vivocomparison of 2′-O-methyl phosphorothioate and morpholino antisense oligonucleotides for Duchenne muscular dystrophy exon skipping

(2009) Hans A. Heemskerk et al.   JOURNAL OF GENE MEDICINE

Prevention of Dystrophic Pathology in Severely Affected Dystrophin/Utrophin-deficient Mice by Morpholino-oligomer-mediated Exon-skipping

(2009) Aurélie Goyenvalle et al.   MOLECULAR THERAPY

Understanding biophysicochemical interactions at the nano–bio interface

(2009) Andre E. Nel et al.   NATURE MATERIALS

A deficit of brain dystrophin impairs specific amygdala GABAergic transmission and enhances defensive behaviour in mice

(2008) Masayuki Sekiguchi et al.   BRAIN

Emergent Dilated Cardiomyopathy Caused by Targeted Repair of Dystrophic Skeletal Muscle

(2008) DeWayne Townsend et al.   MOLECULAR THERAPY

Dystrophin-deficient cardiomyopathy in mouse: Expression of Nox4 and Lox are associated with fibrosis and altered functional parameters in the heart

(2008) Christopher F. Spurney et al.   NEUROMUSCULAR DISORDERS