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Outputs (63)

GENERATION OF THREE-DIMENSIONAL HUMAN ARTIFICIAL SKELETAL MUSCLE TISSUE FROM IPS CELLS ENABLES COMPLEX DISEASE MODELLING FOR MUSCULAR DYSTROPHY
Presentation / Conference Contribution
Maffioletti, S. M., Sarcar, S., Henderson, A., Mannhardt, I., Pinton, L., Moyle, L. A., Steele-Stallard, H., Cappellari, O., Wells, K. E., Ragazzi, M., Wang, W., Zammit, P., Wells, D. J., Eschenhagen, T., & Tedesco, F. S. GENERATION OF THREE-DIMENSIONAL HUMAN ARTIFICIAL SKELETAL MUSCLE TISSUE FROM IPS CELLS ENABLES COMPLEX DISEASE MODELLING FOR MUSCULAR DYSTROPHY

Determination of qPCR Reference Genes Suitable for Normalizing Gene Expression in a Canine Model of Duchenne Muscular Dystrophy
Journal Article
Hildyard, J. C. W., Taylor-Brown, F. E., Massey, C., Wells, D. J., & Piercy, R. J. (in press). Determination of qPCR Reference Genes Suitable for Normalizing Gene Expression in a Canine Model of Duchenne Muscular Dystrophy. Journal of Neuromuscular Diseases, 5(2), 177-191. https://doi.org/10.3233/JND-170267

Background:Dogs with dystrophin-deficient muscular dystrophy are valuable models of the equivalent human disease, Duchenne Muscular Dystrophy (DMD): unlike the mdx mouse, these animals present a disease severity and progression that closely matches t... Read More about Determination of qPCR Reference Genes Suitable for Normalizing Gene Expression in a Canine Model of Duchenne Muscular Dystrophy.

Characterisation of the pathogenic effects of the in vivo expression of an ALS-linked mutation in D-amino acid oxidase: Phenotype and loss of spinal cord motor neurons
Journal Article
Kondori, N. R., Paul, P., Robbins, J. P., Liu, K., Hildyard, J. C. W., Wells, D. J., & De Belleroche, J. S. (in press). Characterisation of the pathogenic effects of the in vivo expression of an ALS-linked mutation in D-amino acid oxidase: Phenotype and loss of spinal cord motor neurons. PLoS ONE, 12, e0188912. https://doi.org/10.1371/journal.pone.0188912

Amyotrophic lateral sclerosis (ALS) is the most common adult-onset neuromuscular disorder characterised by selective loss of motor neurons leading to fatal paralysis. Current therapeutic approaches are limited in their effectiveness. Substantial adva... Read More about Characterisation of the pathogenic effects of the in vivo expression of an ALS-linked mutation in D-amino acid oxidase: Phenotype and loss of spinal cord motor neurons.

THE PHYSIOLOGICAL CONSEQUENCES OF DIFFERENT LEVELS OF DYSTROPHIN FOLLOWING ANTISENSE BASED EXON-SKIPPING IN THE MDX MOUSE
Presentation / Conference Contribution
Muses, S., Godfrey, C., McClorey, G., Wells, K. E., Coursindel, T., Terry, R. L., Betts, C., Cappellari, O., Hammond, S., O'Donovan, E., Hildyard, J. C. W., El Andaloussi, S., Gait, M., Wood, M., & Wells, D. J. THE PHYSIOLOGICAL CONSEQUENCES OF DIFFERENT LEVELS OF DYSTROPHIN FOLLOWING ANTISENSE BASED EXON-SKIPPING IN THE MDX MOUSE

We examined the effects on muscle physiology of restoring different levels of dystrophin in mdx mice with established dystrophic pathophysiology (12 weeks and older). Dystrophin expression was induced very efficiently using cell penetrating peptides... Read More about THE PHYSIOLOGICAL CONSEQUENCES OF DIFFERENT LEVELS OF DYSTROPHIN FOLLOWING ANTISENSE BASED EXON-SKIPPING IN THE MDX MOUSE.

The TREAT-NMD advisory committee for therapeutics (TACT): an innovative de-risking model to foster orphan drug development
Journal Article
Heslop, E., Csimma, C., Straub, V., McCall, J., Nagaraju, K., Wagner, K. R., Caizergues, D., Korinthenberg, R., Flanigan, K. M., Kaufmann, P., McNeil, E., Mendell, J., Hesterlee, S., & Wells, D. J. (in press). The TREAT-NMD advisory committee for therapeutics (TACT): an innovative de-risking model to foster orphan drug development. Orphanet Journal of Rare Diseases, 10(49), https://doi.org/10.1186/s13023-015-0258-1

THE TREAT-NMD ADVISORY COMMITTEE FOR THERAPEUTICS (TACT): AN INNOVATIVE DE-RISKING MODEL TO FOSTER ORPHAN DRUG DEVELOPMENT
Presentation / Conference Contribution
Wells, D. J., Heslop, E., Csimma, C., Straub, V., McCall, J., Nagaraju, K., Wagner, K., Caizergues, D., Korinthenberg, R., Flanigan, K., Mendell, J., Kelly, M., Kaufmann, P., McNeil, E., Robertson, A., Johnston, L., & Bushby, K. THE TREAT-NMD ADVISORY COMMITTEE FOR THERAPEUTICS (TACT): AN INNOVATIVE DE-RISKING MODEL TO FOSTER ORPHAN DRUG DEVELOPMENT