Preclinical Studies for Gene Therapy of Duchenne Muscular Dystrophy

Muscling in: Gene therapies for muscular dystrophy target RNA

(2010) Joel R Chamberlain et al.   NATURE MEDICINE

Limb-girdle muscular dystrophy type 2D gene therapy restores α-sarcoglycan and associated proteins

(2009) Jerry R. Mendell et al.   ANNALS OF NEUROLOGY

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

Emerging strategies for cell and gene therapy of the muscular dystrophies

(2009) Lindsey A. Muir et al.   EXPERT REVIEWS IN MOLECULAR MEDICINE

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

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

Truncated dystrophins can influence neuromuscular synapse structure

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

Evaluation of Vascular Delivery Methodologies to Enhance rAAV6-mediated Gene Transfer to Canine Striated Musculature

(2009) Paul Gregorevic et al.   MOLECULAR THERAPY

Immune Responses to AAV in Canine Muscle Monitored by Cellular Assays and Noninvasive Imaging

(2009) Zejing Wang et al.   MOLECULAR THERAPY

Prevention of Dystrophic Pathology in Severely Affected Dystrophin/Utrophin-deficient Mice by Morpholino-oligomer-mediated Exon-skipping

(2009) Aurélie Goyenvalle et al.   MOLECULAR THERAPY

Persistent Expression of FLAG-tagged Micro dystrophin in Nonhuman Primates Following Intramuscular and Vascular Delivery

(2009) Louise R Rodino-Klapac et al.   MOLECULAR THERAPY

Molecular and cellular adaptations to chronic myotendinous strain injury in mdx mice expressing a truncated dystrophin

(2008) Glen B. Banks et al.   HUMAN MOLECULAR GENETICS

Safety and Efficacy of Regional Intravenous (RI) Versus Intramuscular (IM) Delivery of rAAV1 and rAAV8 to Nonhuman Primate Skeletal Muscle

(2008) Alice Toromanoff et al.   MOLECULAR THERAPY

Percutaneous Transendocardial Delivery of Self-complementary Adeno-associated Virus 6 Achieves Global Cardiac Gene Transfer in Canines

(2008) Lawrence T Bish 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

Recombinant Adeno-associated Virus Transduction and Integration

(2008) Brian R Schultz et al.   MOLECULAR THERAPY

Transduction Efficiency and Immune Response Associated With the Administration of AAV8 Vector Into Dog Skeletal Muscle

(2008) Sachiko Ohshima et al.   MOLECULAR THERAPY