Emerging strategies for cell and gene therapy of the muscular dystrophies

A Renaissance for Antisense Oligonucleotide Drugs in Neurology

(2009) Toshifumi Yokota et al.   ARCHIVES OF NEUROLOGY

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

Octa-guanidine Morpholino Restores Dystrophin Expression in Cardiac and Skeletal Muscles and Ameliorates Pathology in Dystrophic mdx Mice

(2009) Bo Wu et al.   MOLECULAR THERAPY

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

Chromosome number variation in three mouse embryonic stem cell lines during culture

(2008) Paola Rebuzzini et al.   CYTOTECHNOLOGY

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

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

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

Context matters: In vivo and in vitro influences on muscle satellite cell activity

(2008) D.D.W. Cornelison   JOURNAL OF CELLULAR BIOCHEMISTRY

By-passing the nonsense mutation in the 4CVmouse model of muscular dystrophy by induced exon skipping

(2008) Chalermchai Mitrpant et al.   JOURNAL OF GENE MEDICINE

Infectious Molecular Clones of Adeno-Associated Virus Isolated Directly from Human Tissues

(2008) B. C. Schnepp et al.   JOURNAL OF VIROLOGY

Human ESCs predisposition to karyotypic instability: Is a matter of culture adaptation or differential vulnerability among hESC lines due to inherent properties?

(2008) Puri Catalina et al.   Molecular Cancer

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

Emergent Dilated Cardiomyopathy Caused by Targeted Repair of Dystrophic Skeletal Muscle

(2008) DeWayne Townsend et al.   MOLECULAR THERAPY

Recombinant Adeno-associated Virus Transduction and Integration

(2008) Brian R Schultz 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

Another New “Super Muscle Stem Cell” Leaves Unaddressed the Real Problems of Cell Therapy for Duchenne Muscular Dystrophy

(2008) Jacques P Tremblay et al.   MOLECULAR THERAPY

Sarcolemma-localized nNOS is required to maintain activity after mild exercise

(2008) Yvonne M. Kobayashi et al.   NATURE

Functional skeletal muscle regeneration from differentiating embryonic stem cells

(2008) Radbod Darabi et al.   NATURE MEDICINE

Improved cell-penetrating peptide–PNA conjugates for splicing redirection in HeLa cells and exon skipping in mdx mouse muscle

(2008) Gabriela D. Ivanova et al.   NUCLEIC ACIDS RESEARCH

Targeted Chromosomal Insertion of Large DNA into the Human Genome by a Fiber-Modified High-Capacity Adenovirus-Based Vector System

(2008) Manuel A. F. V. Gonçalves et al.   PLoS One

Cell penetrating peptide conjugates of steric block oligonucleotides

(2007) Bernard Lebleu et al.   ADVANCED DRUG DELIVERY REVIEWS

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

(2007) Arkasubhra Ghosh et al.   MOLECULAR THERAPY