Local restoration of dystrophin expression with the morpholino oligomer AVI-4658 in Duchenne muscular dystrophy: a single-blind, placebo-controlled, dose-esclation, proof-of-concept study
(2009)
Journal Article
Kinali, M., Arechavala-Gomeza, V., Feng, L., Cirak, S., Hunt, D., Adkin, C., Guglieri, M., Ashton, E., Abbs, S., Nihoyannopoulos, P., Garralda, M. E., Rutherford, M., McCulley, C., Popplewell, L., Graham, I. R., Dickson, G., Wood, M. J., Wells, D. J., Wilton, S. D., Kole, R., …Muntoni, F. (2009). Local restoration of dystrophin expression with the morpholino oligomer AVI-4658 in Duchenne muscular dystrophy: a single-blind, placebo-controlled, dose-esclation, proof-of-concept study. https://doi.org/10.1016/S1474-4422%2809%2970211-X
Mice that overexpresses human heat shock protein 27 have increased renal injury following ischemia reperfusion
Journal Article
Chen, S. W. C., Kim, M., Kim, M., Song, J. H., Park, S. W., Wells, D. J., De Belleroche, J., D'Agati, V. C., & Lee, H. T. Mice that overexpresses human heat shock protein 27 have increased renal injury following ischemia reperfusion. Kidney International, 75(5), 499-510. https://doi.org/10.1038/ki.2008.572
Chronic Systemic Therapy With Low-dose Morpholino Oligomers Ameliorates the Pathology and Normalizes Locomotor Behavior in mdx Mice
Journal Article
Malerba, A., Graham, I., Sharp, P., Popplewell, L., Foster, K., Wells, D. J., & Dickson, G. Chronic Systemic Therapy With Low-dose Morpholino Oligomers Ameliorates the Pathology and Normalizes Locomotor Behavior in mdx Mice. Molecular Therapy, 19(2), 345-54. https://doi.org/10.1038/mt.2010.261
In vivo fluorescence lifetime tomography of a FRET probe expressed in mouse
Journal Article
McGinty, J., Stuckey, D. W., Solovjev, V. Y., Wylezinska-Arridge, M., Wells, D. J., Arridge, S. R., P M W, F., Hajnal, J. V., & Sardini, A. In vivo fluorescence lifetime tomography of a FRET probe expressed in mouse. Biomedical Optics Express, 2(7), 1907-1917. https://doi.org/10.1364/BOE.2.001907
Muscular dystrophies due to glycosylation defects: diagnosis and therapeutic strategies
Journal Article
Muntoni, F., Torelli, S., Wells, D. J., & Brown, S. C. Muscular dystrophies due to glycosylation defects: diagnosis and therapeutic strategies. Current Opinion in Neurology, 24(5), 437-442. https://doi.org/10.1097/WCO.0b013e32834a95e3
ARE LARGE-IN-FRAME DUPLICATIONS OF DYSTROPHIN FUNCTIONAL?
Presentation / Conference Contribution
Muses, S., Jones, H., & Wells, D. J. ARE LARGE-IN-FRAME DUPLICATIONS OF DYSTROPHIN FUNCTIONAL?
Genetically modified animals and pharmacological research
Book Chapter
Wells, D. J. Genetically modified animals and pharmacological research. . https://doi.org/10.1007/978-3-642-10324-7_9
MAKING SENSE IN DUCHENNE MUSCULAR DYSTROPHY
Presentation / Conference Contribution
Wells, D. J. MAKING SENSE IN DUCHENNE MUSCULAR DYSTROPHY
MOVING FORWARD WITH TACT: 2 YEARS ON
Presentation / Conference Contribution
Heslop, E., Bushby, K., Csimma, C., Straub, V., & Wells, D. J. MOVING FORWARD WITH TACT: 2 YEARS ON
Metformin Treatment Has No Beneficial Effect in a Dose-Response Survival Study in the SOD1(G93A) Mouse Model of ALS and Is Harmful in Female Mice
Journal Article
Kaneb, H. M., Sharp, P. S., Rahmani-Kondori, N., & Wells, D. J. Metformin Treatment Has No Beneficial Effect in a Dose-Response Survival Study in the SOD1(G93A) Mouse Model of ALS and Is Harmful in Female Mice. PLoS ONE, 6(9), e24189. https://doi.org/10.1371/journal.pone.0024189
EXON SKIPPING AND DYSTROPHIN RESTORATION IN DUCHENNE MUSCULAR DYSTROPHY PATIENTS AFTER SYSTEMIC PHOSPHORODIAMIDATE MORPHOLINO OLIGOMER TREATMENT
Presentation / Conference Contribution
Cirak, S., Arechavala-Gomeza, V., Guglieri, M., Feng, L., Torelli, S., Anthony, K., Garralda, M. E., Wells, D. J., Dickson, G., Wood, M. J. A., Wilton, S. D., Straub, V., Shrewsbury, S. B., Sewry, C., Morgan, J. E., Bushby, K., & Muntoni, F. EXON SKIPPING AND DYSTROPHIN RESTORATION IN DUCHENNE MUSCULAR DYSTROPHY PATIENTS AFTER SYSTEMIC PHOSPHORODIAMIDATE MORPHOLINO OLIGOMER TREATMENT
Exon skipping and dystrophin restoration in patients with Duchenne muscular dystrophy after systemic phosphorodiamidate morpholino oligomer treatment: an open-label, phase 2, dose-esclation study
Journal Article
Cirak, S., Arechavala-Gomeza, V., Guglieri, M., Feng, L., Torelli, S., Anthony, K., Abbs, S., Garralda, M. E., Wells, D., Dickson, G., Wood, M. J. A., Wilton, S. D., Straub, V., Shrewsbury, S., Sewry, C., Morgan, J. E., Bushby, K., & Muntoni, F. Exon skipping and dystrophin restoration in patients with Duchenne muscular dystrophy after systemic phosphorodiamidate morpholino oligomer treatment: an open-label, phase 2, dose-esclation study. https://doi.org/10.1016/S0140-6736%2811%2960756-3
Molecular Mechanism of the E99K Mutation in Cardiac Actin (ACTC Gene) That Causes Apical Hypertrophy in Man and Mouse
Journal Article
Song, W., Dyer, E., Stuckey, D. J., Copeland, O., Leung, M. C., Bayliss, C., Messer, A., Wilkinson, R., Tremoleda, J. L., Schneider, M. D., Harding, S. E., Redwood, C. S., Clarke, K., Nowak, K., Monserrat, L., Wells, D. J., & Marston, S. B. (in press). Molecular Mechanism of the E99K Mutation in Cardiac Actin (ACTC Gene) That Causes Apical Hypertrophy in Man and Mouse. Journal of Biological Chemistry, 286(31), 27582–27593. https://doi.org/10.1074/jbc.M111.252320
ASSESSING THE LONG TERM EXPRESSION OF LARGE AS A POTENTIAL THERAPY IN A MOUSE MODEL OF LGMD2I
Presentation / Conference Contribution
Whitmore, C., Booler, H., Parr, C., Ackroyd, M. R., Fernandez-Fuente, M., Muntoni, F., Wells, D., & Brown, S. C. ASSESSING THE LONG TERM EXPRESSION OF LARGE AS A POTENTIAL THERAPY IN A MOUSE MODEL OF LGMD2I
OVER EXPRESSION OF THE LARGE TRANSGENE EXACERBATES MUSCLE PATHOLOGY IN THE MDX MOUSE
Presentation / Conference Contribution
Wells, K. E., Muses, S., Ruberg, A., Terry, R., Shihab, N. K., & Wells, D. J. OVER EXPRESSION OF THE LARGE TRANSGENE EXACERBATES MUSCLE PATHOLOGY IN THE MDX MOUSE
TREATMENT WITH ORAL METFORMIN IMPROVES MUSCLE FUNCTION IN MDX MICE
Presentation / Conference Contribution
Terry, R. L., & Wells, D. J. TREATMENT WITH ORAL METFORMIN IMPROVES MUSCLE FUNCTION IN MDX MICE
POLOXOMER 188 HAS A DELETERIOUS EFFECT ON SKELETAL MUSCLE FUNCTION IN THE MDX MOUSE
Presentation / Conference Contribution
Terry, R. L., Kaneb, H. M., & Wells, D. J. POLOXOMER 188 HAS A DELETERIOUS EFFECT ON SKELETAL MUSCLE FUNCTION IN THE MDX MOUSE
INVESTIGATING THE EFFECTS OF DAO TRANSGENES ON THE SOD1(G93A) MOUSE MODEL OF AMYOTROPHIC LATERAL SCLEROSIS (ALS)
Presentation / Conference Contribution
Rahmani-Kondori, N., de Belleroche, J., & Wells, D. J. INVESTIGATING THE EFFECTS OF DAO TRANSGENES ON THE SOD1(G93A) MOUSE MODEL OF AMYOTROPHIC LATERAL SCLEROSIS (ALS)
RESTORING THE READING FRAME IN LARGE DMD DUPLICATION MUTATIONS RESULTS IN DYSTROPHIN EXPRESSION IN VIVO
Presentation / Conference Contribution
Muses, S., Jones, H., & Wells, D. J. RESTORING THE READING FRAME IN LARGE DMD DUPLICATION MUTATIONS RESULTS IN DYSTROPHIN EXPRESSION IN VIVO
Translational and Regulatory Challenges for Exon Skipping Therapies
Journal Article
Aartsma-Rus, A., Ferlini, A., Goemans, N., Pasmooij, A. M. G., Wells, D. J., Bushby, K., Vroom, E., & Balabanov, P. Translational and Regulatory Challenges for Exon Skipping Therapies. Human Gene Therapy, 25(10), 885-892. https://doi.org/10.1089/hum.2014.086