Animal models of Duchenne muscular dystrophy: from basic mechanisms to gene therapy

JW McGreevy, CH Hakim… - Disease models & …, 2015 - journals.biologists.com
Duchenne muscular dystrophy (DMD) is a progressive muscle-wasting disorder. It is caused
by loss-of-function mutations in the dystrophin gene. Currently, there is no cure. A highly …

Functional correction in mouse models of muscular dystrophy using exon-skipping tricyclo-DNA oligomers

A Goyenvalle, G Griffith, A Babbs, SE Andaloussi… - Nature medicine, 2015 - nature.com
Antisense oligonucleotides (AONs) hold promise for therapeutic correction of many genetic
diseases via exon skipping, and the first AON-based drugs have entered clinical trials for …

Cognitive dysfunction in Duchenne muscular dystrophy: a possible role for neuromodulatory immune molecules

MG Rae, D O'Malley - Journal of neurophysiology, 2016 - journals.physiology.org
Duchenne muscular dystrophy (DMD) is an X chromosome-linked disease characterized by
progressive physical disability, immobility, and premature death in affected boys. Underlying …

[HTML][HTML] Brain Dp140 alters glutamatergic transmission and social behaviour in the mdx52 mouse model of Duchenne muscular dystrophy

Y Hashimoto, H Kuniishi, K Sakai, Y Fukushima… - Progress in …, 2022 - Elsevier
Duchenne muscular dystrophy (DMD) is a muscle disorder caused by DMD mutations and is
characterized by neurobehavioural comorbidities due to dystrophin deficiency in the brain …

Relationships linking emotional, motor, cognitive and GABAergic dysfunctions in dystrophin-deficient mdx mice

C Vaillend, R Chaussenot - Human molecular genetics, 2017 - academic.oup.com
Alterations in the Duchenne muscular dystrophy (DMD) gene have been associated with
enhanced stress reactivity in vertebrate species, suggesting a role for brain dystrophin in …

The unconditioned fear response in dystrophin-deficient mice is associated with adrenal and vascular function

A Lindsay, AP Russell - Scientific Reports, 2023 - nature.com
Loss of function mutations in the gene encoding dystrophin elicits a hypersensitive fear
response in mice and humans. In the dystrophin-deficient mdx mouse, this behaviour is …

Impact of distinct dystrophin gene mutations on behavioral phenotypes of Duchenne muscular dystrophy

A Saoudi, MD Mitsogiannis, F Zarrouki… - Disease Models & …, 2024 - journals.biologists.com
The severity of brain comorbidities in Duchenne muscular dystrophy (DMD) depends on the
mutation position within the DMD gene and differential loss of distinct brain dystrophin …

Social stress is lethal in the mdx model of Duchenne muscular dystrophy

M Razzoli, A Lindsay, ML Law, CM Chamberlain… - …, 2020 - thelancet.com
Background Duchenne muscular dystrophy (DMD) is caused by the loss of dystrophin.
Severe and ultimately lethal, DMD progresses relatively slowly in that patients become …

Long-term efficacy of AAV9-U7snRNA-mediated exon 51 skipping in mdx52 mice

P Aupy, F Zarrouki, Q Sandro, C Gastaldi… - … Therapy Methods & …, 2020 - cell.com
Gene therapy and antisense approaches hold promise for the treatment of Duchenne
muscular dystrophy (DMD). The advantages of both therapeutic strategies can be combined …

Altered social behavior and ultrasonic communication in the dystrophin-deficient mdx mouse model of Duchenne muscular dystrophy

R Miranda, F Nagapin, B Bozon, S Laroche, T Aubin… - Molecular autism, 2015 - Springer
Abstract Background The Duchenne and Becker muscular dystrophies (DMD, BMD) show
significant comorbid diagnosis for autism, and the genomic sequences encoding the …