Therapeutic approaches for Duchenne muscular dystrophy
TC Roberts, MJA Wood, KE Davies - Nature Reviews Drug Discovery, 2023 - nature.com
Duchenne muscular dystrophy (DMD) is a monogenic muscle-wasting disorder and a
priority candidate for molecular and cellular therapeutics. Although rare, it is the most …
priority candidate for molecular and cellular therapeutics. Although rare, it is the most …
Muscle spindle function in healthy and diseased muscle
S Kröger, B Watkins - Skeletal Muscle, 2021 - Springer
Almost every muscle contains muscle spindles. These delicate sensory receptors inform the
central nervous system (CNS) about changes in the length of individual muscles and the …
central nervous system (CNS) about changes in the length of individual muscles and the …
Muscle and cardiac therapeutic strategies for Duchenne muscular dystrophy: past, present, and future
A Łoboda, J Dulak - Pharmacological Reports, 2020 - Springer
Background Duchenne muscular dystrophy (DMD) is a severe X-linked neuromuscular
childhood disorder that causes progressive muscle weakness and degeneration and results …
childhood disorder that causes progressive muscle weakness and degeneration and results …
CRISPR technologies for the treatment of Duchenne muscular dystrophy
E Choi, T Koo - Molecular therapy, 2021 - cell.com
The emerging clustered regularly interspaced short palindromic repeats (CRISPR)-mediated
genome editing technologies have progressed remarkably in recent years, opening up the …
genome editing technologies have progressed remarkably in recent years, opening up the …
Epigenetic modifications in muscle regeneration and progression of Duchenne muscular dystrophy
A Rugowska, A Starosta, P Konieczny - Clinical epigenetics, 2021 - Springer
Duchenne muscular dystrophy (DMD) is a multisystemic disorder that affects 1: 5000 boys.
The severity of the phenotype varies dependent on the mutation site in the DMD gene and …
The severity of the phenotype varies dependent on the mutation site in the DMD gene and …
CRISPR-editing therapy for Duchenne Muscular Dystrophy
F Chemello, EN Olson, R Bassel-Duby - Human gene therapy, 2023 - liebertpub.com
Duchenne muscular dystrophy (DMD) is a debilitating genetic disorder that results in
progressive muscle degeneration and premature death. DMD is caused by mutations in the …
progressive muscle degeneration and premature death. DMD is caused by mutations in the …
Utrophin modulator drugs as potential therapies for Duchenne and Becker muscular dystrophies
P Soblechero‐Martín, A López‐Martínez… - Neuropathology and …, 2021 - Wiley Online Library
Utrophin is an autosomal paralogue of dystrophin, a protein whose deficit causes Duchenne
and Becker muscular dystrophies (DMD/BMD). Utrophin is naturally overexpressed at the …
and Becker muscular dystrophies (DMD/BMD). Utrophin is naturally overexpressed at the …
The multifaceted view of heart problem in Duchenne muscular dystrophy
U Florczyk-Soluch, K Polak, J Dulak - Cellular and Molecular Life Sciences, 2021 - Springer
Dystrophin is a large protein serving as local scaffolding repetitively bridging cytoskeleton
and the outside of striated muscle cell. As such dystrophin is a critical brick primarily in …
and the outside of striated muscle cell. As such dystrophin is a critical brick primarily in …
Evaluating the potential of novel genetic approaches for the treatment of Duchenne muscular dystrophy
V Himič, KE Davies - European Journal of Human Genetics, 2021 - nature.com
Duchenne muscular dystrophy (DMD) is an X-linked progressive muscle-wasting disorder
that is caused by a lack of functional dystrophin, a cytoplasmic protein necessary for the …
that is caused by a lack of functional dystrophin, a cytoplasmic protein necessary for the …
Hydrogen sulfide as a therapeutic option for the treatment of Duchenne muscular dystrophy and other muscle-related diseases
K Kaziród, M Myszka, J Dulak, A Łoboda - Cellular and Molecular Life …, 2022 - Springer
Hydrogen sulfide (H2S) has been known for years as a poisoning gas and until recently
evoked mostly negative associations. However, the discovery of its gasotransmitter functions …
evoked mostly negative associations. However, the discovery of its gasotransmitter functions …