Personal journeys to and in human genetics and dysmorphology.
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Therapeutic approaches for Duchenne muscular dystrophy.
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Utrophin correlates with disease severity in Duchenne muscular dystrophy.
Guiraud S. and Davies K., (2023), Med, 4, 220 - 222
Discovery and mechanism of action studies of 4,6-diphenylpyrimidine-2-carbohydrazides as utrophin modulators for the treatment of Duchenne muscular dystrophy.
Vuorinen A. et al, (2021), Eur J Med Chem, 220
Evaluating the potential of novel genetic approaches for the treatment of Duchenne muscular dystrophy.
Himič V. and Davies KE., (2021), Eur J Hum Genet
Highway to HHGE: An Interview with Dame Kay E. Davies.
Davies K. and Davies KE., (2020), CRISPR J, 3, 325 - 331
Publisher Correction: Therapies for rare diseases: therapeutic modalities, progress and challenges ahead.
Tambuyzer E. et al, (2020), Nat Rev Drug Discov
Therapies for rare diseases: therapeutic modalities, progress and challenges ahead.
Tambuyzer E. et al, (2019), Nature reviews. Drug discovery
Chemical Proteomics and Phenotypic Profiling Identifies the Aryl Hydrocarbon Receptor as a Molecular Target of the Utrophin Modulator Ezutromid.
Wilkinson IVL. et al, (2019), Angew Chem Int Ed Engl
Isolation, structural identification, synthesis, and pharmacological profiling of 1,2-trans-dihydro-1,2-diol metabolites of the utrophin modulator ezutromid
Chatzopoulou M. et al, (2019), Journal of Medicinal Chemistry, 63, 2547 - 2556
Surrogate gene therapy for muscular dystrophy
Davies KE. and Chamberlain JS., (2019), Nature Medicine, 25, 1473 - 1474
Neuronal over-expression of OXR1 is protective against ALS-associated mutant TDP-43 mislocalisation in motor neurons and neuromuscular defects in vivo
Williamson MG. et al, (2019), Human Molecular Genetics
The potential of utrophin and dystrophin combination therapies for Duchenne muscular dystrophy.
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Limitations to adaptive homeostasis in an hyperoxia-induced model of accelerated ageing.
Pomatto LCD. et al, (2019), Redox Biol, 24
Regenerative biomarkers for Duchenne muscular dystrophy
Guiraud S. and Davies KE., (2019), Neural Regeneration Research, 14, 1317 - 1320
Micro-dystrophin Genes Bring Hope of an Effective Therapy for Duchenne Muscular Dystrophy
Davies KE. and Guiraud S., (2019), Molecular therapy : the journal of the American Society of Gene Therapy, 27, 486 - 488
A Phase 1b Trial to Assess the Pharmacokinetics of Ezutromid in Pediatric Duchenne Muscular Dystrophy Patients on a Balanced Diet.
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Embryonic myosin is a regeneration marker to monitor utrophin-based therapies for DMD.
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