All Outputs (25)

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

IMPROVING TRANSLATIONAL STUDIES THROUGH BETTER ANIMAL EXPERIMENTS: LESSONS FROM THE HUMAN RARE DISEASE COMMUNITY
Presentation / Conference Contribution
Wells, D. J. IMPROVING TRANSLATIONAL STUDIES THROUGH BETTER ANIMAL EXPERIMENTS: LESSONS FROM THE HUMAN RARE DISEASE COMMUNITY

MICRO-MINICIRCLE VECTORS FOR SPLICE-SWITCHING IN MOUSE MODEL OF DUCHENNE MUSCULAR DYSTROPHY
Presentation / Conference Contribution
Stenler, S., Andaloussi, S. E. L., Hammond, S., Goyenvalle, A., Wwood, M. J. A., Wells, D. J., Lundin, K. E., Smith, C. I. E., & Blomberg, P. MICRO-MINICIRCLE VECTORS FOR SPLICE-SWITCHING IN MOUSE MODEL OF DUCHENNE MUSCULAR DYSTROPHY

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.

CHARACTERISING THE SKELETAL MUSCLE HISTOLOGICAL PHENOTYPE OF THE DELTAE50-MD DOG, A PRECLINICAL MODEL OF DUCHENNE MUSCULAR DYSTROPHY
Presentation / Conference Contribution
Hildyard, J. C. W., Rawson, F., Harron, R., Riddell, D., Massey, C. A., Taylor-Brown, F. E., Wells, D. J., & Piercy, R. J. CHARACTERISING THE SKELETAL MUSCLE HISTOLOGICAL PHENOTYPE OF THE DELTAE50-MD DOG, A PRECLINICAL MODEL OF DUCHENNE MUSCULAR DYSTROPHY