Low dystrophin levels increase survival and improve muscle pathology and function in dystrophin/utrophin double-knockout mice

Rescue of severely affected dystrophin/utrophin-deficient mice through scAAV-U7snRNA-mediated exon skipping

(2012) Aurélie Goyenvalle et al.   HUMAN MOLECULAR GENETICS

Comparison of skeletal muscle pathology and motor function of dystrophin and utrophin deficient mouse strains

(2012) Maaike van Putten et al.   NEUROMUSCULAR DISORDERS

The Effects of Low Levels of Dystrophin on Mouse Muscle Function and Pathology

(2012) Maaike van Putten et al.   PLoS One

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

Phase 1 Gene Therapy for Duchenne Muscular Dystrophy Using a Translational Optimized AAV Vector

(2011) Dawn E Bowles et al.   MOLECULAR THERAPY

Serum matrix metalloproteinase-9 (MMP-9) as a biomarker for monitoring disease progression in Duchenne muscular dystrophy (DMD)

(2011) V.D. Nadarajah et al.   NEUROMUSCULAR DISORDERS

Systemic Administration of PRO051 in Duchenne's Muscular Dystrophy

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

Diaphragm rescue alone prevents heart dysfunction in dystrophic mice

(2010) Alastair Crisp et al.   HUMAN MOLECULAR GENETICS

A 3 months mild functional test regime does not affect disease parameters in young mdx mice

(2010) Maaike van Putten et al.   NEUROMUSCULAR DISORDERS

Marginal Level Dystrophin Expression Improves Clinical Outcome in a Strain of Dystrophin/Utrophin Double Knockout Mice

(2010) Dejia Li et al.   PLoS One

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

(2009) Aurélie Goyenvalle et al.   MOLECULAR THERAPY

Serum levels of vascular endothelial growth factor elevated in patients with muscular dystrophy

(2008) Toshio Saito et al.   BRAIN & DEVELOPMENT

Disability and survival in Duchenne muscular dystrophy

(2008) M Kohler et al.   JOURNAL OF NEUROLOGY NEUROSURGERY AND PSYCHIATRY

Transduction of Full-length Dystrophin to Multiple Skeletal Muscles Improves Motor Performance and Life Span in Utrophin/Dystrophin Double Knockout Mice

(2008) Ryoko Kawano et al.   MOLECULAR THERAPY