Evaluating the potential of novel genetic approaches for the treatment of Duchenne muscular dystrophy
V Himič, KE Davies - European Journal of Human Genetics, 2021 - nature.com
Duchenne muscular dystrophy (DMD) is an X-linked progressive muscle-wasting disorder
that is caused by a lack of functional dystrophin, a cytoplasmic protein necessary for the …
that is caused by a lack of functional dystrophin, a cytoplasmic protein necessary for the …
Clinical management of Duchenne muscular dystrophy: the state of the art
S Messina, GL Vita - Neurological Sciences, 2018 - Springer
Introduction Duchenne muscular dystrophy (DMD) is a devastating, progressive
neuromuscular disorder for which there is no cure. As the dystrophin gene is located on the …
neuromuscular disorder for which there is no cure. As the dystrophin gene is located on the …
Duchenne muscular dystrophy: an updated review of common available therapies
Background and purpose: Duchenne muscular dystrophy (DMD) is a lethal progressive
pediatric muscle disorder and genetically inherited as an X-linked disease that caused by …
pediatric muscle disorder and genetically inherited as an X-linked disease that caused by …
Innovative therapeutic approaches for Duchenne muscular dystrophy
F Fortunato, R Rossi, MS Falzarano… - Journal of Clinical …, 2021 - mdpi.com
Duchenne muscular dystrophy (DMD) is the most common childhood muscular dystrophy
affecting~ 1: 5000 live male births. Following the identification of pathogenic variations in the …
affecting~ 1: 5000 live male births. Following the identification of pathogenic variations in the …
Combined therapies for Duchenne muscular dystrophy to optimize treatment efficacy
G Cordova, E Negroni, C Cabello-Verrugio… - Frontiers in …, 2018 - frontiersin.org
Duchene Muscular Dystrophy (DMD) is the most frequent muscular dystrophy and one of the
most severe due to the absence of the dystrophin protein. Typical pathological features …
most severe due to the absence of the dystrophin protein. Typical pathological features …
Current pharmacological strategies for Duchenne muscular dystrophy
S Yao, Z Chen, Y Yu, N Zhang, H Jiang… - Frontiers in Cell and …, 2021 - frontiersin.org
Duchenne muscular dystrophy (DMD) is a lethal, X-linked neuromuscular disorder caused
by the absence of dystrophin protein, which is essential for muscle fiber integrity. Loss of …
by the absence of dystrophin protein, which is essential for muscle fiber integrity. Loss of …
Applications of CRISPR/Cas9 for the treatment of Duchenne muscular dystrophy
KRQ Lim, C Yoon, T Yokota - Journal of personalized medicine, 2018 - mdpi.com
Duchenne muscular dystrophy (DMD) is a fatal X-linked recessive neuromuscular disease
prevalent in 1 in 3500 to 5000 males worldwide. As a result of mutations that interrupt the …
prevalent in 1 in 3500 to 5000 males worldwide. As a result of mutations that interrupt the …
Role of CRISPR/Cas9 in the treatment of Duchenne muscular dystrophy and its delivery strategies
Duchenne muscular dystrophy (DMD) is a neuromuscular disorder brought on by mutations
in the DMD gene, which prevent muscle cells from expressing the dystrophin protein …
in the DMD gene, which prevent muscle cells from expressing the dystrophin protein …
Gene therapy for Duchenne muscular dystrophy
IEC Verhaart, A Aartsma-Rus - Current opinion in neurology, 2012 - journals.lww.com
Gene therapy for Duchenne muscular dystrophy : Current Opinion in Neurology Gene therapy
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Impending therapies for Duchenne muscular dystrophy
TA Partridge - Current opinion in neurology, 2011 - journals.lww.com
Although genetic and cell-mediated approaches are currently showing genuine promise in
preclinical and clinical trials, there remains considerable interest in the development of …
preclinical and clinical trials, there remains considerable interest in the development of …