Preclinical Studies in Models of NMD

Preclinical Studies in Models of NMD
Methodology and Characterisation
Elizabeth Greally Magnetic resonance imaging of cardiac function in mouse models for muscular dystrophy associated cardiomyopathy
Tina Duong Mild exercise improves skeletal muscle performance in a mouse model of limb girdle muscular dystrophy 2B 
Raffaella Willmann TREAT-NMD- Activity 07: Accelerate preclinical phase of new therapeutic treatment development 
Shinya Kamauchi Urinary level of prostaglandin D2 metabolites in animal models of Duchenne muscular dystrophy 
Marina Mora An in vitro cell model to further elucidate mechanisms of muscle fibrosis and test possible anti-fibrotic agents 
Bassem F. El-Khodor  Phenotyping Neonatal models of neuromuscular degeneration as a prelude for drug candidate evaluation. 
Miranda Grounds The immediate consequences of treadmill exercise on adult dystrophic mdx mice
Kanneboyina Nagaraju  Preclinical Drug Trial Efforts for Muscular Dystrophy: Methods and End points 
Maaike van Putten  Effect of a mild exercise regime on disease parameters in the mdx mouse model 
Sabine De La Porte MALDI reveals membrane lipid profile reversion in MDX mice 
Joe N. Kornegay Muscle Hypertrophy and Contractures in a Myostatin Heterozygote Null GRMD Dog
Richard Lovering  Structural and functional evaluation of branched myofibers in mdx mice 
Rebecca Pruss  Modelling of SMA in mice - novelties and limits 
David Bendahan  Non invasive Assessment of skeletal muscle function in mouse using 1H-MR imaging and 31P-MR spectroscopy

Antisense approaches in NMD
Peter Sazani  AVI-5038: Initial Efficacy and Safety Evaluation in Cynomolgus Monkeys
Annemieke Aartsma-Rus  Applicability of Antisense-Mediated EXON Skipping for Duchenne Muscular Dystrophy Mutations
Peter 't'Hoen  Dual exon skipping in myostatin and dystrophin as a potential therapy for Duchenne muscular dystrophy 
Pietro Spitali  Guidelines for exon skipping quantification in the DMD gene 
Annemieke Aartsma-Rus  Long Term Systemic Antisense-Mediated Exon Skipping in Dystrophic Mouse Models 
Linda Popplewell  Multiexon skipping in Duchenne muscular dystrophy 
Alberto Malerba  Chronic long term administration of low and high doses of phosphorodiamidate morpholino oligomer ameliorates the dystrophin phenotype in mdx mice 
Dr Matthew JA Wood Muscle and Heart Targeted Splice Correction for DMD 
Adrian Krainer  Antisense Correction of SMN2 Splicing in the CNS of Mouse Models of Spinal Muscular Atrophy 
Peter Sazani  Repeat Dose Mechanistic Toxicology Evaluation of AVI-4225 PMO in mdx mice 
Peter Sazani  Repeat Dose Toxicology Evaluation of AVI-4658 PMO in mdx mice 
Peter Sazani  Repeat Dose Toxicology Evaluation of AVI-4658 PMO in Cynomolgus Monkeys 
Sue Fletcher and Steve Wilton Exon Skipping for Non-Deletion Dystrophin Mutations
Sue Fletcher Induced non-productive splicing to study Muscle Gene Expression
Takashi Saito Analysis of exons 6 and 8 skipping in cells from an exon-7 deleted DMD patient: direct application of antisense sequences found in study with canine muscular dystrophy.

Cell and gene therapy approaches
Stefanie Schinkel  Efficient systemic AAV9-mediated microdystrophin gene transfer in the mdx heart 
Severine Pouillot Automation of biodistribution study 
Sabrina Sacconi  Cell Therapy for Muscular Dystrophy: CD34 Negative Muscle Derived Cells present High Myogenic and No Adipogenic Potential 
Yvan Torrente  Combining Stem Cells and Exon Skipping Aapproach to Treat Muscular Dystrophies 
Vincenzo Nigro  Gene therapy and glucocorticoids for muscular dystrophy and cardiomyopathy of BIO14.6 hamster 
Seng H. Cheng  Gene Therapy or Antisense Oligonucleotide-mediated Augmentation of SMN Levels for Spinal Muscular Atrophy. 

Therapeutic Targets
Li Niu Developing AMPA Receptor Aptamers as New Drug Candidates for ALS 
Mahasweta Girgenrath  Up-regulation of TGF beta signaling in MDC1A 
Jennifer Lachey  Activin Receptor Type IIB Inhibition Improves Strength and Function of Dystrophic Muscle 
Kosuke Aritake  Chemotherapy of Duchenne muscular dystrophy 
Paolo Bonaldo Genetic and pharmacological inhibition of cyclophilin D rescues mitochondrial defects and prevents muscle apoptosis in collagen VI myopathic mice
Annamaria De Luca Exercised dystrophic mdx mice and specific modulation of disease-sensitive indices by drugs clinically relevant for duchenne muscular dystrophy: effects of a chronic treatment with the angiotensin-converting enzyme inhibitor enalapril 
12 Oct 2010