Impending therapies for Duchenne muscular dystrophy

Membrane Sealant Poloxamer P188 Protects Against Isoproterenol Induced Cardiomyopathy in Dystrophin Deficient Mice

(2011) Christopher F Spurney et al.   BMC Cardiovascular Disorders

Intramuscular cell transplantation as a potential treatment of myopathies: clinical and preclinical relevant data

(2011) Daniel Skuk et al.   EXPERT OPINION ON BIOLOGICAL THERAPY

TAT-μUtrophin mitigates the pathophysiology of dystrophin and utrophin double-knockout mice

(2011) Jarrod A. Call et al.   JOURNAL OF APPLIED PHYSIOLOGY

Unknown

(2011) Jennifer Peterson et al.   MOLECULAR MEDICINE

Systemic Administration of PRO051 in Duchenne's Muscular Dystrophy

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

Daily Treatment with SMTC1100, a Novel Small Molecule Utrophin Upregulator, Dramatically Reduces the Dystrophic Symptoms in the mdx Mouse

(2011) Jonathon M. Tinsley et al.   PLoS One

Chronic administration of membrane sealant prevents severe cardiac injury and ventricular dilatation in dystrophic dogs

(2010) DeWayne Townsend et al.   JOURNAL OF CLINICAL INVESTIGATION

Guanine Analogues Enhance Antisense Oligonucleotide-induced Exon Skipping in Dystrophin Gene In Vitro and In Vivo

(2010) Yihong Hu et al.   MOLECULAR THERAPY

The Status of Exon Skipping as a Therapeutic Approach to Duchenne Muscular Dystrophy

(2010) Qi-Long Lu et al.   MOLECULAR THERAPY

Biglycan recruits utrophin to the sarcolemma and counters dystrophic pathology in mdx mice

(2010) A. R. Amenta et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Valproic Acid Activates the PI3K/Akt/mTOR Pathway in Muscle and Ameliorates Pathology in a Mouse Model of Duchenne Muscular Dystrophy

(2009) Praveen B. Gurpur et al.   AMERICAN JOURNAL OF PATHOLOGY

Efficacy of systemic morpholino exon-skipping in duchenne dystrophy dogs

(2009) Toshifumi Yokota et al.   ANNALS OF NEUROLOGY

Peptide-Morpholino Conjugate: A Promising Therapeutic for Duchenne Muscular Dystrophy

(2009) Hong M. Moulton et al.   Annals of the New York Academy of Sciences

Emerging strategies for cell and gene therapy of the muscular dystrophies

(2009) Lindsey A. Muir et al.   EXPERT REVIEWS IN MOLECULAR MEDICINE

Dose-dependent restoration of dystrophin expression in cardiac muscle of dystrophic mice by systemically delivered morpholino

(2009) B Wu et al.   GENE THERAPY

Theoretic applicability of antisense-mediated exon skipping for Duchenne muscular dystrophy mutations

(2009) Annemieke Aartsma-Rus et al.   HUMAN MUTATION

Osteopontin promotes fibrosis in dystrophic mouse muscle by modulating immune cell subsets and intramuscular TGF-β

(2009) Sylvia A. Vetrone et al.   JOURNAL OF CLINICAL INVESTIGATION

Dystrophins carrying spectrin-like repeats 16 and 17 anchor nNOS to the sarcolemma and enhance exercise performance in a mouse model of muscular dystrophy

(2009) Yi Lai et al.   JOURNAL OF CLINICAL INVESTIGATION

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

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

In Vivo Myogenic Potential of Human CD133+ Muscle-derived Stem Cells: A Quantitative Study

(2009) Elisa Negroni et al.   MOLECULAR THERAPY

Enhanced Exon-skipping Induced by U7 snRNA Carrying a Splicing Silencer Sequence: Promising Tool for DMD Therapy

(2009) Aurélie Goyenvalle et al.   MOLECULAR THERAPY

Functional Substitution by TAT-Utrophin in Dystrophin-Deficient Mice

(2009) Kevin J. Sonnemann et al.   PLOS MEDICINE

Poloxamer 188 reduces the contraction-induced force decline in lumbrical muscles from mdx mice

(2008) Rainer Ng et al.   AMERICAN JOURNAL OF PHYSIOLOGY-CELL PHYSIOLOGY

Highly Efficient, Functional Engraftment of Skeletal Muscle Stem Cells in Dystrophic Muscles

(2008) Massimiliano Cerletti et al.   CELL

IMPLICATIONS OF CROSS-TALK BETWEEN TUMOUR NECROSIS FACTOR AND INSULIN-LIKE GROWTH FACTOR-1 SIGNALLING IN SKELETAL MUSCLE

(2008) Miranda D Grounds et al.   CLINICAL AND EXPERIMENTAL PHARMACOLOGY AND PHYSIOLOGY

Shifts in macrophage phenotypes and macrophage competition for arginine metabolism affect the severity of muscle pathology in muscular dystrophy

(2008) S. A. Villalta et al.   HUMAN MOLECULAR GENETICS

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

(2008) HaiFang Yin et al.   HUMAN MOLECULAR GENETICS

Transient Immunomodulation Allows Repeated Injections of AAV1 and Correction of Muscular Dystrophy in Multiple Muscles

(2008) Stéphanie Lorain et al.   MOLECULAR THERAPY

SJLDystrophic Mice Express a Significant Amount of Human Muscle Proteins Following Systemic Delivery of Human Adipose-Derived Stromal Cells Without Immunosuppression

(2008) Natássia M. Vieira et al.   STEM CELLS