Dual exon skipping in myostatin and dystrophin for Duchenne muscular dystrophy

AAV-directed muscular dystrophy gene therapy

(2010) Ying Tang et al.   EXPERT OPINION ON BIOLOGICAL THERAPY

Coordinated patterns of gene expressions for adult muscle build-up in transgenic mice expressing myostatin propeptide

(2009) Baoping Zhao et al.   BMC GENOMICS

Gene therapy for muscular dystrophy: current progress and future prospects

(2009) Capucine Trollet et al.   EXPERT OPINION ON BIOLOGICAL THERAPY

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

Diagnosis and new treatments in muscular dystrophies

(2009) A Y Manzur et al.   JOURNAL OF NEUROLOGY NEUROSURGERY AND PSYCHIATRY

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

Molecular, cellular and physiological investigation of myostatin propeptide-mediated muscle growth in adult mice

(2009) Antonios Matsakas et al.   NEUROMUSCULAR DISORDERS

RNA-targeting approaches for neuromuscular diseases

(2009) Florence Le Roy et al.   TRENDS IN MOLECULAR MEDICINE

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

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

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

Proteomic Identification and Functional Validation of Activins and Bone Morphogenetic Protein 11 as Candidate Novel Muscle Mass Regulators

(2008) Tatyana A. Souza et al.   MOLECULAR ENDOCRINOLOGY

Guidelines for Antisense Oligonucleotide Design and Insight Into Splice-modulating Mechanisms

(2008) Annemieke Aartsma-Rus et al.   MOLECULAR THERAPY

Single muscle fiber contractile properties in adults with muscular dystrophy treated with MYO-029

(2008) Lisa S. Krivickas et al.   MUSCLE & NERVE

Myostatin signals through Pax7 to regulate satellite cell self-renewal

(2007) Craig McFarlane et al.   EXPERIMENTAL CELL RESEARCH