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 …

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 …

Duchenne muscular dystrophy: an updated review of common available therapies

A Salmaninejad, SF Valilou, H Bayat… - International Journal …, 2018 - Taylor & Francis
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 …

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 …

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 …

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 …

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 …

Role of CRISPR/Cas9 in the treatment of Duchenne muscular dystrophy and its delivery strategies

P Agrawal, V Harish, S Mohd, SK Singh, D Tewari… - Life Sciences, 2023 - Elsevier
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 …

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
for Duchenne muscular dystrophy : Current Opinion in Neurology Log in or Register Subscribe …

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 …