Long-Term Systemic Myostatin Inhibition via Liver-Targeted Gene Transfer in Golden Retriever Muscular Dystrophy

Gene Therapy in Large Animal Models of Muscular Dystrophy

(2013) Z. Wang et al.   ILAR JOURNAL

Targeting the Activin Type IIB Receptor to Improve Muscle Mass and Function in the mdx Mouse Model of Duchenne Muscular Dystrophy

(2011) Emidio E. Pistilli et al.   AMERICAN JOURNAL OF PATHOLOGY

Evaluation of Adeno-Associated Viral Vectors for Liver-Directed Gene Transfer in Dogs

(2011) Peter Bell et al.   HUMAN GENE THERAPY

Changes in muscle T2 and tissue damage after downhill running in mdx Mice

(2011) Sunita Mathur et al.   MUSCLE & NERVE

Morpholinos and their peptide conjugates: Therapeutic promise and challenge for Duchenne muscular dystrophy

(2010) Hong M. Moulton et al.   BIOCHIMICA ET BIOPHYSICA ACTA-BIOMEMBRANES

Gene therapy for muscle disease

(2010) Yuko Miyagoe-Suzuki et al.   EXPERIMENTAL CELL RESEARCH

Read-Through Strategies for Suppression of Nonsense Mutations in Duchenne/ Becker Muscular Dystrophy: Aminoglycosides and Ataluren (PTC124)

(2010) Richard S. Finkel   JOURNAL OF CHILD NEUROLOGY

The Potential of Exon Skipping for Treatment for Duchenne Muscular Dystrophy

(2010) Terence Partridge   JOURNAL OF CHILD NEUROLOGY

Widespread Muscle Expression of an AAV9 Human Mini-dystrophin Vector After Intravenous Injection in Neonatal Dystrophin-deficient Dogs

(2010) Joe N Kornegay et al.   MOLECULAR THERAPY

Activin IIB receptor blockade attenuates dystrophic pathology in a mouse model of duchenne muscular dystrophy

(2010) Kevin J. Morine et al.   MUSCLE & NERVE

Systemic Myostatin Inhibition via Liver-Targeted Gene Transfer in Normal and Dystrophic Mice

(2010) Kevin J. Morine et al.   PLoS One

A Duchenne Muscular Dystrophy Gene Hot Spot Mutation in Dystrophin-Deficient Cavalier King Charles Spaniels Is Amenable to Exon 51 Skipping

(2010) Gemma L. Walmsley et al.   PLoS One

Efficacy of systemic morpholino exon-skipping in duchenne dystrophy dogs

(2009) Toshifumi Yokota et al.   ANNALS OF NEUROLOGY

Hydrodynamic Limb Vein Injection of Adeno-Associated Virus Serotype 8 Vector Carrying Canine Myostatin Propeptide Gene into Normal Dogs Enhances Muscle Growth

(2009) Chunping Qiao et al.   HUMAN GENE THERAPY

Persistent Expression of FLAG-tagged Micro dystrophin in Nonhuman Primates Following Intramuscular and Vascular Delivery

(2009) Louise R Rodino-Klapac et al.   MOLECULAR THERAPY

Quantitative MR imaging of individual muscle involvement in facioscapulohumeral muscular dystrophy

(2009) Hermien E. Kan et al.   NEUROMUSCULAR DISORDERS

A phase I/IItrial of MYO-029 in adult subjects with muscular dystrophy

(2008) Kathryn R. Wagner et al.   ANNALS OF NEUROLOGY

Long-Term Benefit of Adeno-Associated Virus/Antisense-Mediated Exon Skipping in Dystrophic Mice

(2008) Michela Alessandra Denti et al.   HUMAN GENE THERAPY

Myostatin Propeptide Gene Delivery by Adeno-Associated Virus Serotype 8 Vectors Enhances Muscle Growth and Ameliorates Dystrophic Phenotypes in mdx Mice

(2008) Chunping Qiao et al.   HUMAN GENE THERAPY

Myostatin Directly Regulates Skeletal Muscle Fibrosis

(2008) Zhao Bo Li et al.   JOURNAL OF BIOLOGICAL CHEMISTRY

Myostatin blockade improves function but not histopathology in a murine model of limb-girdle muscular dystrophy 2C

(2007) Sasha Bogdanovich et al.   MUSCLE & NERVE