[HTML][HTML] Therapeutic strategies for dystrophin replacement in Duchenne muscular dystrophy

C Happi Mbakam, G Lamothe, JP Tremblay - Frontiers in Medicine, 2022 - frontiersin.org
Duchenne muscular dystrophy (DMD) is an X-linked hereditary disease characterized by
progressive muscle wasting due to modifications in the DMD gene (exon deletions …

Facioscapulohumeral muscular dystrophy: genetics, gene activation and downstream signalling with regard to recent therapeutic approaches: an update

T Schätzl, L Kaiser, HP Deigner - Orphanet Journal of Rare Diseases, 2021 - Springer
Whilst a disease-modifying treatment for Facioscapulohumeral muscular dystrophy (FSHD)
does not exist currently, recent advances in complex molecular pathophysiology studies of …

Loss of TDP-43 function and rimmed vacuoles persist after T cell depletion in a xenograft model of sporadic inclusion body myositis

KA Britson, JP Ling, KE Braunstein… - Science translational …, 2022 - science.org
Sporadic inclusion body myositis (IBM) is the most common acquired muscle disease in
adults over age 50, yet it remains unclear whether the disease is primarily driven by T cell …

Complexity of skeletal muscle degeneration: Multi-systems pathophysiology and organ crosstalk in dystrophinopathy

K Ohlendieck, D Swandulla - Pflügers Archiv-European Journal of …, 2021 - Springer
Duchenne muscular dystrophy is a highly progressive muscle wasting disorder due to
primary abnormalities in one of the largest genes in the human genome, the DMD gene …

[HTML][HTML] Structural and functional changes in the coupling of fascial tissue, skeletal muscle, and nerves during aging

A Zullo, J Fleckenstein, R Schleip, K Hoppe… - Frontiers in …, 2020 - frontiersin.org
Aging is a one-way process associated with profound structural and functional changes in
the organism. Indeed, the neuromuscular system undergoes a wide remodeling, which …

Available in vitro models for human satellite cells from skeletal muscle

C Romagnoli, T Iantomasi, ML Brandi - International Journal of Molecular …, 2021 - mdpi.com
Skeletal muscle accounts for almost 40% of the total adult human body mass. This tissue is
essential for structural and mechanical functions such as posture, locomotion, and …

Therapeutic strategies targeting DUX4 in FSHD

L Le Gall, E Sidlauskaite, V Mariot… - Journal of Clinical …, 2020 - mdpi.com
Facioscapulohumeral muscular dystrophy (FSHD) is a common muscle dystrophy typically
affecting patients within their second decade. Patients initially exhibit asymmetric facial and …

Optimization of xenografting methods for generating human skeletal muscle in mice

A O'Neill, AL Martinez, AL Mueller… - Cell …, 2024 - journals.sagepub.com
Xenografts of human skeletal muscle generated in mice can be used to study muscle
pathology and to test drugs designed to treat myopathies and muscular dystrophies for their …

The diversity of muscles and their regenerative potential across animals

L Zullo, M Bozzo, A Daya, A Di Clemente, FP Mancini… - Cells, 2020 - mdpi.com
Cells with contractile functions are present in almost all metazoans, and so are the related
processes of muscle homeostasis and regeneration. Regeneration itself is a complex …

Magnetically-assisted viral transduction (magnetofection) medical applications: An update

B Azadpour, N Aharipour, A Paryab, H Omid… - Biomaterials …, 2023 - Elsevier
Gene therapy involves replacing a faulty gene or adding a new gene inside the body's cells
to cure disease or improve the body's ability to fight disease. Its popularity is evident from …