Therapeutic strategies for Duchenne muscular dystrophy: an update
Neuromuscular disorders encompass a heterogeneous group of conditions that impair the
function of muscles, motor neurons, peripheral nerves, and neuromuscular junctions. Being …
function of muscles, motor neurons, peripheral nerves, and neuromuscular junctions. Being …
Overview of gene therapy in spinal muscular atrophy and Duchenne muscular dystrophy
NJ Abreu, MA Waldrop - Pediatric pulmonology, 2021 - Wiley Online Library
Both 5q‐linked spinal muscular atrophy (SMA) and Duchenne muscular dystrophy (DMD)
are fatal monogenic neuromuscular disorders caused by loss‐of‐function mutations. SMA is …
are fatal monogenic neuromuscular disorders caused by loss‐of‐function mutations. SMA is …
[HTML][HTML] Advance trends in targeting homology-directed repair for accurate gene editing: An inclusive review of small molecules and modified CRISPR-Cas9 systems
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Introduction: Clustered regularly interspaced short palindromic repeat and its associated …
Introduction: Clustered regularly interspaced short palindromic repeat and its associated …
A combined prospective and retrospective comparison of long-term functional outcomes suggests delayed loss of ambulation and pulmonary decline with long-term …
O Mitelman, HZ Abdel-Hamid, BJ Byrne… - Journal of …, 2022 - content.iospress.com
Background: Studies 4658-201/202 (201/202) evaluated treatment effects of eteplirsen over
4 years in patients with Duchenne muscular dystrophy and confirmed exon-51 amenable …
4 years in patients with Duchenne muscular dystrophy and confirmed exon-51 amenable …
The dystrophin node as integrator of cytoskeletal organization, lateral force transmission, fiber stability and cellular signaling in skeletal muscle
P Dowling, S Gargan, S Murphy, M Zweyer, H Sabir… - Proteomes, 2021 - mdpi.com
The systematic bioanalytical characterization of the protein product of the DMD gene, which
is defective in the pediatric disorder Duchenne muscular dystrophy, led to the discovery of …
is defective in the pediatric disorder Duchenne muscular dystrophy, led to the discovery of …
Anti-RANKL therapy prevents glucocorticoid-induced bone loss and promotes muscle function in a mouse model of Duchenne muscular dystrophy
Bisphosphonates prevent bone loss in glucocorticoid (GC)-treated boys with Duchenne
muscular dystrophy (DMD) and are recommended as standard of care. Targeting receptor …
muscular dystrophy (DMD) and are recommended as standard of care. Targeting receptor …
[HTML][HTML] Duchenne's muscular dystrophy involves a defective transsulfuration pathway activity
Duchenne muscular dystrophy (DMD) is the most frequent X chromosome-linked disease
caused by mutations in the gene encoding for dystrophin, leading to progressive and …
caused by mutations in the gene encoding for dystrophin, leading to progressive and …
Evaluation of cardiac function in young Mdx mice using MRI with feature tracking and self-gated magnetic resonance cine imaging
J Ueda, S Saito - Diagnostics, 2023 - mdpi.com
This study aimed to evaluate cardiac function in a young mouse model of Duchenne
muscular dystrophy (mdx) using cardiac magnetic resonance imaging (MRI) with feature …
muscular dystrophy (mdx) using cardiac magnetic resonance imaging (MRI) with feature …
Precision medicine using whole genome sequencing identifies a novel dystrophin (DMD) variant for X‐linked muscular dystrophy in a cat
GD Shelton, F Tucciarone, LT Guo… - Journal of Veterinary …, 2024 - Wiley Online Library
Abstract Background Muscular dystrophies (MDs) are a large, heterogeneous group of
degenerative muscle diseases. X‐linked dystrophin‐deficient MD in cats is the first …
degenerative muscle diseases. X‐linked dystrophin‐deficient MD in cats is the first …
An update on the application of CRISPR technology in clinical practice
F Morshedzadeh, M Ghanei, M Lotfi, M Ghasemi… - Molecular …, 2024 - Springer
The CRISPR/Cas system, an innovative gene-editing tool, is emerging as a promising
technique for genome modifications. This straightforward technique was created based on …
technique for genome modifications. This straightforward technique was created based on …