Gene therapy for Duchenne muscular dystrophy

Full-Length Dystrophin Reconstitution with Adeno-Associated Viral Vectors

(2014) William Lostal et al.   HUMAN GENE THERAPY

Forelimb Treatment in a Large Cohort of Dystrophic Dogs Supports Delivery of a Recombinant AAV for Exon Skipping in Duchenne Patients

(2014) Caroline Le Guiner et al.   MOLECULAR THERAPY

Long-Term Safety and Efficacy of Factor IX Gene Therapy in Hemophilia B

(2014) Amit C. Nathwani et al.   NEW ENGLAND JOURNAL OF MEDICINE

Adeno-associated viral vectors do not efficiently target muscle satellite cells

(2014) Andrea LH Arnett et al.   Molecular Therapy-Methods & Clinical Development

Long-Term Robust Myocardial Transduction of the Dog Heart from a Peripheral Vein by Adeno-Associated Virus Serotype-8

(2013) Xiufang Pan et al.   HUMAN GENE THERAPY

Triple Trans-Splicing Adeno-Associated Virus Vectors Capable of Transferring the Coding Sequence for Full-Length Dystrophin Protein into Dystrophic Mice

(2013) Taeyoung Koo et al.   HUMAN GENE THERAPY

CpG-depleted adeno-associated virus vectors evade immune detection

(2013) Susan M. Faust et al.   JOURNAL OF CLINICAL INVESTIGATION

Microdystrophin Ameliorates Muscular Dystrophy in the Canine Model of Duchenne Muscular Dystrophy

(2013) Jin-Hong Shin et al.   MOLECULAR THERAPY

Plasmapheresis Eliminates the Negative Impact of AAV Antibodies on Microdystrophin Gene Expression Following Vascular Delivery

(2013) LG Chicoine et al.   MOLECULAR THERAPY

Plasmid-Encoded Proinsulin Preserves C-Peptide While Specifically Reducing Proinsulin-Specific CD8+ T Cells in Type 1 Diabetes

(2013) B. O. Roep et al.   Science Translational Medicine

Sarcospan: a small protein with large potential for Duchenne muscular dystrophy

(2013) Jamie L Marshall et al.   Skeletal Muscle

Manufacturing and Regulatory Strategies for Clinical AAV2-hRPE65

(2012) J. Fraser Wright et al.   CURRENT GENE THERAPY

MyD88 Signaling in B Cells Regulates the Production of Th1-dependent Antibodies to AAV

(2012) Muriel Sudres et al.   MOLECULAR THERAPY

Successful Regional Delivery and Long-term Expression of a Dystrophin Gene in Canine Muscular Dystrophy: A Preclinical Model for Human Therapies

(2012) Zejing Wang et al.   MOLECULAR THERAPY

 2 and  3 helices of dystrophin R16 and R17 frame a microdomain in the  1 helix of dystrophin R17 for neuronal NOS binding

(2012) Y. Lai et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

The genome of self-complementary adeno-associated viral vectors increases Toll-like receptor 9-dependent innate immune responses in the liver

(2011) A. T. Martino et al.   BLOOD

Successful attenuation of humoral immunity to viral capsid and transgenic protein following AAV-mediated gene transfer with a non-depleting CD4 antibody and cyclosporine

(2011) J H McIntosh et al.   GENE THERAPY

Lateral transmission of force is impaired in skeletal muscles of dystrophic mice and very old rats

(2011) Krishnan S. Ramaswamy et al.   JOURNAL OF PHYSIOLOGY-LONDON

Capsid-specific T-cell Responses to Natural Infections With Adeno-associated Viruses in Humans Differ From Those of Nonhuman Primates

(2011) Hua Li et al.   MOLECULAR THERAPY

Immunity and AAV-Mediated Gene Therapy for Muscular Dystrophies in Large Animal Models and Human Trials

(2011) Zejing Wang et al.   Frontiers in Microbiology

Innate Immune Responses to AAV Vectors

(2011) Geoffrey L. Rogers et al.   Frontiers in Microbiology

Sustained alpha-sarcoglycan gene expression after gene transfer in limb-girdle muscular dystrophy, type 2D

(2010) Jerry R. Mendell et al.   ANNALS OF 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

Adeno-associated Virus Vectors Serotype 2 Induce Prolonged Proliferation of Capsid-Specific CD8+ T Cells in Mice

(2010) Hua Li et al.   MOLECULAR THERAPY

Gene Therapy of mdx Mice With Large Truncated Dystrophins Generated by Recombination Using rAAV6

(2010) Guy L Odom et al.   MOLECULAR THERAPY

The Complex and Evolving Story of T cell Activation to AAV Vector-encoded Transgene Products

(2010) Lauren E Mays et al.   MOLECULAR THERAPY

Dystrophin Immunity in Duchenne's Muscular Dystrophy

(2010) Jerry R. Mendell et al.   NEW ENGLAND JOURNAL OF MEDICINE

AAV-1-mediated gene transfer to skeletal muscle in humans results in dose-dependent activation of capsid-specific T cells

(2009) F. Mingozzi et al.   BLOOD

Dystrophin is a microtubule-associated protein

(2009) Kurt W. Prins et al.   JOURNAL OF CELL BIOLOGY

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

Capsid antigen presentation flags human hepatocytes for destruction after transduction by adeno-associated viral vectors

(2009) Gary C. Pien et al.   JOURNAL OF CLINICAL INVESTIGATION

The TLR9-MyD88 pathway is critical for adaptive immune responses to adeno-associated virus gene therapy vectors in mice

(2009) Jiangao Zhu et al.   JOURNAL OF CLINICAL INVESTIGATION

Adeno-Associated Virus Capsid Structure Drives CD4-Dependent CD8 + T Cell Response to Vector Encoded Proteins

(2009) Lauren E. Mays et al.   JOURNAL OF IMMUNOLOGY

An Ankyrin-Based Mechanism for Functional Organization of Dystrophin and Dystroglycan

(2008) Gai Ayalon et al.   CELL

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

(2008) Brian Bostick et al.   HUMAN GENE THERAPY

Diverse IgG subclass responses to adeno-associated virus infection and vector administration

(2008) Samuel L. Murphy et al.   JOURNAL OF MEDICAL VIROLOGY

Complement Is an Essential Component of the Immune Response to Adeno-Associated Virus Vectors

(2008) A. K. Zaiss et al.   JOURNAL OF VIROLOGY

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

(2008) Stéphanie Lorain et al.   MOLECULAR THERAPY

A Single Intravenous Injection of Adeno-associated Virus Serotype-9 Leads to Whole Body Skeletal Muscle Transduction in Dogs

(2008) Yongping Yue 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

Differential targeting of nNOS and AQP4 to dystrophin-deficient sarcolemma by membrane-directed  -dystrobrevin

(2007) M. E. Adams et al.   JOURNAL OF CELL SCIENCE