Duchenne muscular dystrophy: Disease mechanism and therapeutic strategies

A Bez Batti Angulski, N Hosny, H Cohen… - Frontiers in …, 2023 - frontiersin.org
Duchenne muscular dystrophy (DMD) is a severe, progressive, and ultimately fatal disease
of skeletal muscle wasting, respiratory insufficiency, and cardiomyopathy. The identification …

Emerging therapies for Duchenne muscular dystrophy

T Markati, M Oskoui, MA Farrar, T Duong… - The Lancet …, 2022 - thelancet.com
Duchenne muscular dystrophy is an X-linked disease caused by the absence of functional
dystrophin in the muscle cells. Major advances have led to the development of gene …

Histological methods to assess skeletal muscle degeneration and regeneration in Duchenne muscular dystrophy

N Dubuisson, R Versele, C Planchon… - International journal of …, 2022 - mdpi.com
Duchenne muscular dystrophy (DMD) is a progressive disease caused by the loss of
function of the protein dystrophin. This protein contributes to the stabilisation of striated cells …

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 …

Internal ribosome entry site (IRES)-mediated translation and its potential for novel mRNA-based therapy development

R Marques, R Lacerda, L Romão - Biomedicines, 2022 - mdpi.com
Many conditions can benefit from RNA-based therapies, namely, those targeting internal
ribosome entry sites (IRESs) and their regulatory proteins, the IRES trans-acting factors …

Histone deacetylases: Molecular mechanisms and therapeutic implications for muscular dystrophies

M Sandonà, G Cavioli, A Renzini, A Cedola… - International Journal of …, 2023 - mdpi.com
Histone deacetylases (HDACs) are enzymes that regulate the deacetylation of numerous
histone and non-histone proteins, thereby affecting a wide range of cellular processes …

Integrated Comparative Transcriptome and circRNA-lncRNA-miRNA-mRNA ceRNA Regulatory Network Analyses Identify Molecular Mechanisms Associated with …

V Dehghanian Reyhan, F Ghafouri, M Sadeghi… - Animals, 2023 - mdpi.com
Simple Summary Meat quality and human-health-related indexes are important traits in beef
cattle breeding. Intramuscular fat content (IMF) is a major meat quality trait that influences …

Advances in CRISPR/Cas9 genome editing for the treatment of muscular dystrophies

S Fatehi, RM Marks, MJ Rok, L Perillat… - Human Gene …, 2023 - liebertpub.com
Muscular dystrophies (MDs) comprise a diverse group of inherited disorders characterized
by progressive muscle loss and weakness. Given the genetic etiology underlying MDs …

[HTML][HTML] Lessons learned from discontinued clinical developments in Duchenne muscular dystrophy

T Markati, L De Waele, U Schara-Schmidt… - Frontiers in …, 2021 - frontiersin.org
Duchenne muscular dystrophy (DMD) is an X-linked condition caused by a deficiency of
functional dystrophin protein. Patients experience progressive muscle weakness …

Sarcoplasmic Reticulum Ca2+ Buffer Proteins: A Focus on the Yet-To-Be-Explored Role of Sarcalumenin in Skeletal Muscle Health and Disease

E Conte, G Dinoi, P Imbrici, A De Luca, A Liantonio - Cells, 2023 - mdpi.com
Sarcalumenin (SAR) is a luminal Ca2+ buffer protein with high capacity but low affinity for
calcium binding found predominantly in the longitudinal sarcoplasmic reticulum (SR) of fast …