Inhibition of antigen presentation during AAV gene therapy using virus peptides

Progress toward Gene Therapy for Duchenne Muscular Dystrophy

(2017) Joel R. Chamberlain et al.   MOLECULAR THERAPY

Muscle-specific CRISPR/Cas9 dystrophin gene editing ameliorates pathophysiology in a mouse model for Duchenne muscular dystrophy

(2017) Niclas E. Bengtsson et al.   Nature Communications

Overcoming the Host Immune Response to Adeno-Associated Virus Gene Delivery Vectors: The Race Between Clearance, Tolerance, Neutralization, and Escape

(2017) Federico Mingozzi et al.   Annual Review of Virology

Adeno-Associated Virus (AAV) Mediated Dystrophin Gene Transfer Studies and Exon Skipping Strategies for Duchenne Muscular Dystrophy (DMD)

(2015) Klaudia Kawecka et al.   CURRENT GENE THERAPY

Safe and bodywide muscle transduction in young adult Duchenne muscular dystrophy dogs with adeno-associated virus

(2015) Yongping Yue et al.   HUMAN MOLECULAR GENETICS

Postnatal genome editing partially restores dystrophin expression in a mouse model of muscular dystrophy

(2015) C. Long et al.   SCIENCE

In vivo gene editing in dystrophic mouse muscle and muscle stem cells

(2015) M. Tabebordbar et al.   SCIENCE

In vivo genome editing improves muscle function in a mouse model of Duchenne muscular dystrophy

(2015) C. E. Nelson et al.   SCIENCE

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

Immune Responses to Intramuscular Administration of Alipogene Tiparvovec (AAV1-LPLS447X) in a Phase II Clinical Trial of Lipoprotein Lipase Deficiency Gene Therapy

(2013) Valerie Ferreira et al.   HUMAN GENE THERAPY

Human Treg responses allow sustained recombinant adeno-associated virus–mediated transgene expression

(2013) Christian Mueller et al.   JOURNAL OF CLINICAL INVESTIGATION

Adeno-associated virus capsid antigen presentation is dependent on endosomal escape

(2013) Chengwen Li et al.   JOURNAL OF CLINICAL INVESTIGATION

Oversized AAV Transductifon Is Mediated via a DNA-PKcs-independent, Rad51C-dependent Repair Pathway

(2013) Matthew L Hirsch et al.   MOLECULAR THERAPY

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

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

AAV8 Induces Tolerance in Murine Muscle as a Result of Poor APC Transduction, T Cell Exhaustion, and Minimal MHCI Upregulation on Target Cells

(2013) Lauren E Mays 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

A Simplified Immune Suppression Scheme Leads to Persistent Micro-dystrophin Expression in Duchenne Muscular Dystrophy Dogs

(2011) Jin-Hong Shin et al.   HUMAN GENE THERAPY

Phase 1 Gene Therapy for Duchenne Muscular Dystrophy Using a Translational Optimized AAV Vector

(2011) Dawn E Bowles et al.   MOLECULAR THERAPY

Adenovirus-Associated Virus Vector–Mediated Gene Transfer in Hemophilia B

(2011) Amit C. Nathwani et al.   NEW ENGLAND JOURNAL OF MEDICINE

The human cytomegalovirus microRNA miR-UL112 acts synergistically with a cellular microRNA to escape immune elimination

(2010) Daphna Nachmani et al.   NATURE IMMUNOLOGY

Dystrophin Immunity in Duchenne's Muscular Dystrophy

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

Systemic Trans-Splicing Adeno-Associated Viral Delivery Efficiently Transduces the Heart of Adult mdx Mouse, a Model for Duchenne Muscular Dystrophy

(2009) Arkasubhra Ghosh et al.   HUMAN GENE THERAPY

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

Cytotoxic-T-Lymphocyte-Mediated Elimination of Target Cells Transduced with Engineered Adeno-Associated Virus Type 2 Vector In Vivo

(2009) C. Li et al.   JOURNAL OF VIROLOGY

Evidence for the Failure of Adeno-associated Virus Serotype 5 to Package a Viral Genome ≥8.2 kb

(2009) Yi Lai et al.   MOLECULAR THERAPY

Cellular immune response to cryptic epitopes during therapeutic gene transfer

(2009) C. Li et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Loading of oncolytic vesicular stomatitis virus onto antigen-specific T cells enhances the efficacy of adoptive T-cell therapy of tumors

(2008) J Qiao et al.   GENE THERAPY

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

(2008) Brian Bostick et al.   HUMAN GENE THERAPY

Systemic humanminidystrophingene transfer improves functions and life span of dystrophin and dystrophin/utrophin-deficient mice

(2008) Bing Wang et al.   JOURNAL OF ORTHOPAEDIC RESEARCH

Structural and Functional Dissection of the Human Cytomegalovirus Immune Evasion Protein US6

(2008) G. E. Dugan 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

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

(2008) Guy L Odom et al.   MOLECULAR THERAPY