Challenges associated with homologous directed repair using CRISPR-Cas9 and TALEN to edit the DMD genetic mutation in canine Duchenne muscular dystrophy
S Mata López, C Balog-Alvarez, S Vitha, AK Bettis… - PloS one, 2020 - journals.plos.org
Duchenne muscular dystrophy (DMD) is caused by mutations in the DMD gene that abolish
the expression of dystrophin protein. Dogs with the genetic homologue, golden retriever …
the expression of dystrophin protein. Dogs with the genetic homologue, golden retriever …
[PDF][PDF] Diverse effector and regulatory functions of fibro/adipogenic progenitors during skeletal muscle fibrosis in muscular dystrophy
X Wang, J Chen, ST Homma, Y Wang, GR Smith… - Iscience, 2023 - cell.com
Fibrosis is a prominent pathological feature of skeletal muscle in Duchenne muscular
dystrophy (DMD). The commonly used disease mouse model, mdx 5cv, displays progressive …
dystrophy (DMD). The commonly used disease mouse model, mdx 5cv, displays progressive …
Proteomic identification of markers of membrane repair, regeneration and fibrosis in the aged and dystrophic diaphragm
Deficiency in the membrane cytoskeletal protein dystrophin is the underlying cause of the
progressive muscle wasting disease named Duchenne muscular dystrophy. In order to …
progressive muscle wasting disease named Duchenne muscular dystrophy. In order to …
Myostatin and activin blockade by engineered follistatin results in hypertrophy and improves dystrophic pathology in mdx mouse more than myostatin blockade alone
A Iskenderian, N Liu, Q Deng, Y Huang, C Shen… - Skeletal Muscle, 2018 - Springer
Background Myostatin antagonists are being developed as therapies for Duchenne
muscular dystrophy due to their strong hypertrophic effects on skeletal muscle. Engineered …
muscular dystrophy due to their strong hypertrophic effects on skeletal muscle. Engineered …
ActRIIB: ALK4-Fc alleviates muscle dysfunction and comorbidities in murine models of neuromuscular disorders
J Li, M Fredericks, M Cannell, K Wang… - The Journal of …, 2021 - Am Soc Clin Investig
Patients with neuromuscular disorders suffer from a lack of treatment options for skeletal
muscle weakness and disease comorbidities. Here, we introduce as a potential therapeutic …
muscle weakness and disease comorbidities. Here, we introduce as a potential therapeutic …
Mechanically patterned neuromuscular junctions-in-a-dish have improved functional maturation
CL Happe, KP Tenerelli, AK Gromova… - Molecular biology of …, 2017 - Am Soc Cell Biol
Motor neuron (MN) diseases are progressive disorders resulting from degeneration of
neuromuscular junctions (NMJs), which form the connection between MNs and muscle …
neuromuscular junctions (NMJs), which form the connection between MNs and muscle …
[HTML][HTML] Safety, tolerability and pharmacokinetics of eteplirsen in young boys aged 6–48 months with Duchenne muscular dystrophy amenable to exon 51 skipping
E Mercuri, AM Seferian, L Servais, N Deconinck… - Neuromuscular …, 2023 - Elsevier
Eteplirsen is FDA-approved for the treatment of Duchenne muscular dystrophy (DMD) in
exon 51 skip-amenable patients. Previous studies in boys> 4 years of age indicate …
exon 51 skip-amenable patients. Previous studies in boys> 4 years of age indicate …
Progressive skeletal muscle atrophy in muscular dystrophies: a role for toll-like receptor-signaling in disease pathogenesis
B De Paepe - International Journal of Molecular Sciences, 2020 - mdpi.com
Muscle atrophy is an active process controlled by specific transcriptional programs, in which
muscle mass is lost by increased protein degradation and/or decreased protein synthesis …
muscle mass is lost by increased protein degradation and/or decreased protein synthesis …
Calcium handling dysfunction and cardiac damage following acute ventricular preload challenge in the dystrophin-deficient mouse heart
V Haffner, Z Nourian, EM Boerman… - American Journal …, 2023 - journals.physiology.org
Duchenne muscular dystrophy (DMD) is the most common muscular dystrophy and is
caused by mutations in the dystrophin gene. Dystrophin deficiency is associated with …
caused by mutations in the dystrophin gene. Dystrophin deficiency is associated with …
RhoA/ROCK2 signalling is enhanced by PDGF‐AA in fibro‐adipogenic progenitor cells: implications for Duchenne muscular dystrophy
E Fernández‐Simón, X Suárez‐Calvet… - Journal of Cachexia …, 2022 - Wiley Online Library
Background The lack of dystrophin expression in Duchenne muscular dystrophy (DMD)
induces muscle fibre and replacement by fibro‐adipose tissue. Although the role of some …
induces muscle fibre and replacement by fibro‐adipose tissue. Although the role of some …