Evaluation of an AAV9-mini-dystrophin gene therapy candidate in a rat model of Duchenne muscular dystrophy

Cardiovascular phenotype of the Dmdmdx rat – a suitable animal model for Duchenne muscular dystrophy

(2021) Petra Lujza Szabó et al.   Disease Models & Mechanisms

Duchenne muscular dystrophy

(2021) Dongsheng Duan et al.   Nature Reviews Disease Primers

Dystrophin and mini-dystrophin quantification by mass spectrometry in skeletal muscle for gene therapy development in Duchenne muscular dystrophy

(2021) Vahid Farrokhi et al.   GENE THERAPY

TRPC3, but not TRPC1, as a good therapeutic target for standalone or complementary treatment of DMD

(2021) Anna Creisméas et al.   Journal of Translational Medicine

Characterization of brain dystrophins absence and impact in dystrophin-deficient Dmdmdx rat model

(2020) Dorian Caudal et al.   PLoS One

NATURAL HISTORY OF SERUM ENZYME LEVELS IN DUCHENNE MUSCULAR DYSTROPHY AND IMPLICATIONS FOR CLINICAL PRACTICE

(2020) Maricela Rodríguez-Cruz et al.   AMERICAN JOURNAL OF PHYSICAL MEDICINE & REHABILITATION

Restorative treatments of dystrophin expression in Duchenne muscular dystrophy: A systematic review

(2020) Carlos Pascual‐Morena et al.   Annals of Clinical and Translational Neurology

Development of Novel Micro-dystrophins with Enhanced Functionality

(2019) Julian N. Ramos et al.   MOLECULAR THERAPY

Duchenne muscular dystrophy: an historical treatment review

(2019) Lineu Cesar Werneck et al.   ARQUIVOS DE NEURO-PSIQUIATRIA

Immunophenotype of a Rat Model of Duchenne's Disease and Demonstration of Improved Muscle Strength After Anti-CD45RC Antibody Treatment

(2019) Laure-Hélène Ouisse et al.   Frontiers in Immunology

Micro-dystrophin Gene Therapy Partially Enhances Exercise Capacity in Older Adult mdx Mice

(2019) Buel D. Rodgers et al.   Molecular Therapy-Methods & Clinical Development

Long-term effects of glucocorticoids on function, quality of life, and survival in patients with Duchenne muscular dystrophy: a prospective cohort study

(2018) Craig M McDonald et al.   LANCET

Systemic AAV micro-dystrophin gene therapy for Duchenne muscular dystrophy

(2018) Dongsheng Duan   MOLECULAR THERAPY

Exhaustive characterization of the newly developed Duchenne muscular dystrophy rat model: a unique animal model for DMD which mimics the human disease at both the muscular and the cardiac levels

(2017) C. Huchet et al.   NEUROMUSCULAR DISORDERS

Long-term microdystrophin gene therapy is effective in a canine model of Duchenne muscular dystrophy

(2017) Caroline Le Guiner et al.   Nature Communications

Direct interaction of human serum proteins with AAV virions to enhance AAV transduction: immediate impact on clinical applications

(2016) M Wang et al.   GENE THERAPY

Manufacturing of recombinant adeno-associated viral vectors for clinical trials

(2016) Nathalie Clément et al.   Molecular Therapy-Methods & Clinical Development

Animal models of Duchenne muscular dystrophy: from basic mechanisms to gene therapy

(2015) J. W. McGreevy et al.   Disease Models & Mechanisms

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

(2015) Yongping Yue et al.   HUMAN MOLECULAR GENETICS

AAV-8 Is More Efficient than AAV-9 in Transducing Neonatal Dog Heart

(2015) Xiufang Pan et al.   Human Gene Therapy Methods

Characterization of Dystrophin Deficient Rats: A New Model for Duchenne Muscular Dystrophy

(2014) Thibaut Larcher et al.   PLoS One

Generation of muscular dystrophy model rats with a CRISPR/Cas system

(2014) Katsuyuki Nakamura et al.   Scientific Reports

Report of MDA muscle disease symposium on newborn screening for Duchenne muscular dystrophy

(2013) Jerry R. Mendell et al.   MUSCLE & NERVE

Structural Insight into the Unique Properties of Adeno-Associated Virus Serotype 9

(2012) M. A. DiMattia et al.   JOURNAL OF VIROLOGY

Comparison of Adeno-Associated Virus Serotypes and Delivery Methods for Cardiac Gene Transfer

(2012) Hongfei Fang et al.   Human Gene Therapy Methods

TerminalN-Linked Galactose Is the Primary Receptor for Adeno-associated Virus 9

(2011) Shen Shen et al.   JOURNAL OF BIOLOGICAL CHEMISTRY

Engineering Liver-detargeted AAV9 Vectors for Cardiac and Musculoskeletal Gene Transfer

(2011) Nagesh Pulicherla 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

Application of WGA lectin staining for visualization of the connective tissue in skeletal muscle, bone, and ligament/tendon studies

(2010) Tatiana Y. Kostrominova   MICROSCOPY RESEARCH AND TECHNIQUE

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

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

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 (AAV) Serotype 9 Provides Global Cardiac Gene Transfer Superior to AAV1, AAV6, AAV7, and AAV8 in the Mouse and Rat

(2008) Lawrence T. Bish 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

Analysis of AAV Serotypes 1–9 Mediated Gene Expression and Tropism in Mice After Systemic Injection

(2008) Carmela Zincarelli et al.   MOLECULAR THERAPY