How much dystrophin is enough: the physiological consequences of different levels of dystrophin in themdxmouse

Delivery of therapeutic oligonucleotides with cell penetrating peptides

(2015) Prisca Boisguérin et al.   ADVANCED DRUG DELIVERY REVIEWS

Exon skipping restores dystrophin expression, but fails to prevent disease progression in later stage dystrophic dko mice

(2014) B Wu et al.   GENE THERAPY

Poloxomer 188 Has a Deleterious Effect on Dystrophic Skeletal Muscle Function

(2014) Rebecca L. Terry et al.   PLoS One

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

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

VBP15, a novel anti-inflammatory and membrane-stabilizer, improves muscular dystrophy without side effects

(2013) Christopher R. Heier et al.   EMBO Molecular Medicine

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

(2013) Maaike van Putten et al.   FASEB JOURNAL

The transgenic expression of LARGE exacerbates the muscle phenotype of dystroglycanopathy mice

(2013) Charlotte Whitmore et al.   HUMAN MOLECULAR GENETICS

Dystromirs as Serum Biomarkers for Monitoring the Disease Severity in Duchenne Muscular Dystrophy

(2013) Irina T. Zaharieva et al.   PLoS One

Preventing phosphorylation of dystroglycan ameliorates the dystrophic phenotype in mdx mouse

(2012) Gaynor Miller et al.   HUMAN MOLECULAR GENETICS

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

(2012) Maaike van Putten et al.   PLoS One

Expression Analysis in Multiple Muscle Groups and Serum Reveals Complexity in the MicroRNA Transcriptome of the mdx Mouse with Implications for Therapy

(2012) Thomas C Roberts et al.   Molecular Therapy-Nucleic Acids

Pip6-PMO, A New Generation of Peptide-oligonucleotide Conjugates With Improved Cardiac Exon Skipping Activity for DMD Treatment

(2012) Corinne Betts et al.   Molecular Therapy-Nucleic Acids

Dystrophin quantification and clinical correlations in Becker muscular dystrophy: implications for clinical trials

(2011) K. Anthony et al.   BRAIN

miR-31 modulates dystrophin expression: new implications for Duchenne muscular dystrophy therapy

(2011) Davide Cacchiarelli et al.   EMBO REPORTS

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

Targeting RNA to treat neuromuscular disease

(2011) Francesco Muntoni et al.   NATURE REVIEWS DRUG DISCOVERY

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

Preclinical PK and PD Studies on 2′-O-Methyl-phosphorothioate RNA Antisense Oligonucleotides in the mdx Mouse Model

(2010) Hans Heemskerk et al.   MOLECULAR THERAPY

In-frame Dystrophin Following Exon 51-Skipping Improves Muscle Pathology and Function in the Exon 52–Deficient mdx Mouse

(2010) Yoshitsugu Aoki et al.   MOLECULAR THERAPY

Functional Rescue of Dystrophin-deficient mdx Mice by a Chimeric Peptide-PMO

(2010) HaiFang Yin et al.   MOLECULAR THERAPY

Physiological Characterization of Muscle Strength With Variable Levels of Dystrophin Restoration in mdx Mice Following Local Antisense Therapy

(2010) Paul S Sharp et al.   MOLECULAR THERAPY

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

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

(2010) Dejia Li et al.   PLoS One

A fusion peptide directs enhanced systemic dystrophin exon skipping and functional restoration in dystrophin-deficient mdx mice

(2009) HaiFang Yin et al.   HUMAN MOLECULAR GENETICS

Local restoration of dystrophin expression with the morpholino oligomer AVI-4658 in Duchenne muscular dystrophy: a single-blind, placebo-controlled, dose-escalation, proof-of-concept study

(2009) Maria Kinali et al.   LANCET NEUROLOGY

Immunohistological intensity measurements as a tool to assess sarcolemma-associated protein expression

(2009) V. Arechavala-Gomeza et al.   NEUROPATHOLOGY AND APPLIED NEUROBIOLOGY

Preservation of Muscle Force in Mdx3cv Mice Correlates with Low-Level Expression of a Near Full-Length Dystrophin Protein

(2008) Dejia Li et al.   AMERICAN JOURNAL OF PATHOLOGY

Update on the management of Duchenne muscular dystrophy

(2008) A Y Manzur et al.   ARCHIVES OF DISEASE IN CHILDHOOD

Cell-penetrating peptide-conjugated antisense oligonucleotides restore systemic muscle and cardiac dystrophin expression and function

(2008) HaiFang Yin et al.   HUMAN MOLECULAR GENETICS

Codon and mRNA Sequence Optimization of Microdystrophin Transgenes Improves Expression and Physiological Outcome in Dystrophic mdx Mice Following AAV2/8 Gene Transfer

(2008) Helen Foster et al.   MOLECULAR THERAPY

Sustained Dystrophin Expression Induced by Peptide-conjugated Morpholino Oligomers in the Muscles of mdx Mice

(2008) Natee Jearawiriyapaisarn et al.   MOLECULAR THERAPY