Gene therapy for muscular dystrophy: current progress and future prospects

Inhibiting Myostatin with Follistatin Improves the Success of Myoblast Transplantation in Dystrophic Mice

(2009) Basma F. Benabdallah et al.   CELL TRANSPLANTATION

A soluble activin type IIB receptor improves function in a mouse model of amyotrophic lateral sclerosis

(2009) Brett M. Morrison et al.   EXPERIMENTAL 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

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

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

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

Truncated dystrophins can influence neuromuscular synapse structure

(2009) Glen B. Banks et al.   MOLECULAR AND CELLULAR NEUROSCIENCE

Design of Phosphorodiamidate Morpholino Oligomers (PMOs) for the Induction of Exon Skipping of the Human DMD Gene

(2009) Linda J Popplewell et al.   MOLECULAR THERAPY

Inhibition of myostatin with emphasis on follistatin as a therapy for muscle disease

(2009) Louise R. Rodino-Klapac et al.   MUSCLE & NERVE

Disease Rescue and Increased Lifespan in a Model of Cardiomyopathy and Muscular Dystrophy by Combined AAV Treatments

(2009) Carmen Vitiello et al.   PLoS One

Adeno-Associated Virus-8-Mediated Intravenous Transfer of Myostatin Propeptide Leads to Systemic Functional Improvements of Slow but Not Fast Muscle

(2009) Keith Foster et al.   REJUVENATION RESEARCH

Overexpression of Galgt2 in skeletal muscle prevents injury resulting from eccentric contractions in both mdx and wild-type mice

(2008) Paul T. Martin et al.   AMERICAN JOURNAL OF PHYSIOLOGY-CELL PHYSIOLOGY

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

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

Investigating the pathology of Emery–Dreifuss muscular dystrophy

(2008) Susan C. Brown et al.   BIOCHEMICAL SOCIETY TRANSACTIONS

Molecular signatures of Emery–Dreifuss muscular dystrophy

(2008) Matthew A. Wheeler et al.   BIOCHEMICAL SOCIETY TRANSACTIONS

Assessment of the feasibility of exon 45–55 multiexon skipping for duchenne muscular dystrophy

(2008) Laura van Vliet et al.   BMC Medical Genetics

Myotonic dystrophy protein kinase (DMPK) and its role in the pathogenesis of myotonic dystrophy 1

(2008) Perla Kaliman et al.   CELLULAR SIGNALLING

Oculopharyngeal muscular dystrophy: A polyalanine myopathy

(2008) Bernard Brais   Current Neurology and Neuroscience Reports

Combining stem cells and exon skipping strategy to treat muscular dystrophy

(2008) Mirella Meregalli et al.   EXPERT OPINION ON BIOLOGICAL THERAPY

Correction of dystrophia myotonica type 1 pre-mRNA transcripts by artificial trans-splicing

(2008) H Y Chen et al.   GENE THERAPY

Atelocollagen-mediated local and systemic applications of myostatin-targeting siRNA increase skeletal muscle mass

(2008) N Kinouchi et al.   GENE THERAPY

Construction and analysis of compact muscle-specific promoters for AAV vectors

(2008) B Wang et al.   GENE THERAPY

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

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

Protein Trans-Splicing as a Means for Viral Vector-Mediated In Vivo Gene Therapy

(2008) Juan Li et al.   HUMAN GENE THERAPY

Adeno-Associated Virus Serotype-9 Microdystrophin Gene Therapy Ameliorates Electrocardiographic Abnormalities in mdx Mice

(2008) Brian Bostick 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

Cell-lineage regulated myogenesis for dystrophin replacement: a novel therapeutic approach for treatment of muscular dystrophy

(2008) E. Kimura et al.   HUMAN MOLECULAR GENETICS

Myostatin Directly Regulates Skeletal Muscle Fibrosis

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

Genetic Complementation of Human Muscle Cells via Directed Stem Cell Fusion

(2008) Manuel AFV Gonçalves et al.   MOLECULAR THERAPY

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

Microutrophin Delivery Through rAAV6 Increases Lifespan and Improves Muscle Function in Dystrophic Dystrophin/Utrophin-deficient Mice

(2008) Guy L Odom et al.   MOLECULAR THERAPY

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

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

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

PlGF–MMP-9–expressing cells restore microcirculation and efficacy of cell therapy in aged dystrophic muscle

(2008) Cesare Gargioli et al.   NATURE MEDICINE

Calpain 3, the “gatekeeper” of proper sarcomere assembly, turnover and maintenance

(2008) Jacques S. Beckmann et al.   NEUROMUSCULAR DISORDERS

Muscular dystrophies due to glycosylation defects

(2008) Francesco Muntoni et al.   Neurotherapeutics

Therapeutic possibilities in the autosomal recessive limb-girdle muscular dystrophies

(2008) Volker Straub et al.   Neurotherapeutics

Long-term enhancement of skeletal muscle mass and strength by single gene administration of myostatin inhibitors

(2008) A. M. Haidet et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Transgenic expression of a myostatin inhibitor derived from follistatin increases skeletal muscle mass and ameliorates dystrophic pathology in mdx mice

(2007) Masashi Nakatani et al.   FASEB JOURNAL

Lentivirus Mediated HO-1 Gene Transfer Enhances Myogenic Precursor Cell Survival After Autologous Transplantation in Pig

(2007) Thomas Laumonier et al.   MOLECULAR THERAPY

A Hybrid Vector System Expands Adeno-associated Viral Vector Packaging Capacity in a Transgene-independent Manner

(2007) Arkasubhra Ghosh et al.   MOLECULAR THERAPY

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

(2007) Sasha Bogdanovich et al.   MUSCLE & NERVE