Developing DMD therapeutics: a review of the effectiveness of small molecules, stop-codon readthrough, dystrophin gene replacement, and exon-skipping therapies
O Sheikh, T Yokota - Expert opinion on investigational drugs, 2021 - Taylor & Francis
Introduction Duchenne muscular dystrophy (DMD) is an X-linked recessive disorder caused
by mutations in the dystrophin (DMD) gene. Most patients die from respiratory failure or …
by mutations in the dystrophin (DMD) gene. Most patients die from respiratory failure or …
All muscle satellite cells are equal, but are some more equal than others?
PS Zammit - Journal of cell science, 2008 - journals.biologists.com
Skeletal muscle is an accessible adult stem-cell model in which differentiated myofibres are
maintained and repaired by a self-renewing stem-cell compartment. These resident stem …
maintained and repaired by a self-renewing stem-cell compartment. These resident stem …
Sustained dystrophin expression induced by peptide-conjugated morpholino oligomers in the muscles of mdx mice
N Jearawiriyapaisarn, HM Moulton, B Buckley… - Molecular Therapy, 2008 - cell.com
Cell-penetrating peptides (CPPs), containing arginine (R), 6-aminohexanoic acid (X), and/or
β-alanine (B) conjugated to phosphorodiamidate morpholino oligomers (PMOs), enhance …
β-alanine (B) conjugated to phosphorodiamidate morpholino oligomers (PMOs), enhance …
Histone deacetylases: Molecular mechanisms and therapeutic implications for muscular dystrophies
Histone deacetylases (HDACs) are enzymes that regulate the deacetylation of numerous
histone and non-histone proteins, thereby affecting a wide range of cellular processes …
histone and non-histone proteins, thereby affecting a wide range of cellular processes …
Muscle diseases: the muscular dystrophies
EM McNally, P Pytel - Annu. Rev. Pathol. Mech. Dis., 2007 - annualreviews.org
Dystrophic muscle disease can occur at any age. Early-or childhood-onset muscular
dystrophies may be associated with profound loss of muscle function, affecting ambulation …
dystrophies may be associated with profound loss of muscle function, affecting ambulation …
Effects of in vivo injury on the neuromuscular junction in healthy and dystrophic muscles
Key points• Strength loss induced by lengthening contractions is typically attributed to
damaged force‐bearing structures within skeletal muscle. Muscle lacking the structural …
damaged force‐bearing structures within skeletal muscle. Muscle lacking the structural …
Pre-and postsynaptic changes in the neuromuscular junction in dystrophic mice
Duchenne muscular dystrophy (DMD) is a devastating neuromuscular disease in which
weakness, increased susceptibility to muscle injury, and inadequate repair appear to …
weakness, increased susceptibility to muscle injury, and inadequate repair appear to …
The validity of the International Physical Activity Questionnaire (IPAQ) for adults with progressive muscle diseases
SF Roberts-Lewis, CM White, M Ashworth… - Disability and …, 2022 - Taylor & Francis
Purpose Measuring the physical activity of adults with progressive muscle diseases is
important to inform clinical practice, for activity recommendations and for outcomes …
important to inform clinical practice, for activity recommendations and for outcomes …
Recovery of altered neuromuscular junction morphology and muscle function in mdx mice after injury
Duchenne muscular dystrophy (DMD) is a devastating neuromuscular disease in which
weakness, increased susceptibility to muscle injury, and inadequate repair underlie the …
weakness, increased susceptibility to muscle injury, and inadequate repair underlie the …
Therapeutic implications of miRNAs for muscle-wasting conditions
L Yedigaryan, M Sampaolesi - Cells, 2021 - mdpi.com
MicroRNAs (miRNAs) are small, non-coding RNA molecules that are mainly involved in
translational repression by binding to specific messenger RNAs. Recently, miRNAs have …
translational repression by binding to specific messenger RNAs. Recently, miRNAs have …