Duchenne muscular dystrophy

D Duan, N Goemans, S Takeda, E Mercuri… - Nature Reviews …, 2021 - nature.com
Duchenne muscular dystrophy is a severe, progressive, muscle-wasting disease that leads
to difficulties with movement and, eventually, to the need for assisted ventilation and …

Abnormal calcium handling in Duchenne muscular dystrophy: mechanisms and potential therapies

S Mareedu, ED Million, D Duan, GJ Babu - Frontiers in physiology, 2021 - frontiersin.org
Duchenne muscular dystrophy (DMD) is an X-linked muscle-wasting disease caused by the
loss of dystrophin. DMD is associated with muscle degeneration, necrosis, inflammation …

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 …

Ion channels of the sarcolemma and intracellular organelles in Duchenne muscular dystrophy: a role in the dysregulation of ion homeostasis and a possible target for …

MV Dubinin, KN Belosludtsev - International Journal of Molecular …, 2023 - mdpi.com
Duchenne muscular dystrophy (DMD) is caused by the absence of the dystrophin protein
and a properly functioning dystrophin-associated protein complex (DAPC) in muscle cells …

Duchenne muscular dystrophy: pathogenesis and promising therapies

M Chang, Y Cai, Z Gao, X Chen, B Liu, C Zhang… - Journal of …, 2023 - Springer
Duchenne muscular dystrophy (DMD) is a severe, progressive, muscle-wasting disease,
characterized by progressive deterioration of skeletal muscle that causes rapid loss of …

Microproteins: overlooked regulators of physiology and disease

KR Hassel, O Brito-Estrada, CA Makarewich - Iscience, 2023 - cell.com
Ongoing efforts to generate a complete and accurate annotation of the genome have
revealed a significant blind spot for small proteins (< 100 amino acids) originating from short …

The cardiac translational landscape reveals that micropeptides are new players involved in cardiomyocyte hypertrophy

Y Yan, R Tang, B Li, L Cheng, S Ye, T Yang, YC Han… - Molecular Therapy, 2021 - cell.com
Hypertrophic growth of cardiomyocytes is one of the major compensatory responses in the
heart after physiological or pathological stimulation. Protein synthesis enhancement, which …

Duchenne muscular dystrophy gene therapy in 2023: status, perspective, and beyond

D Duan - Human Gene Therapy, 2023 - liebertpub.com
Duchenne muscular dystrophy (DMD) was named more than 150 years ago. About four
decades ago, the DMD gene was discovered, and the reading frame shift was determined …

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 …

Inhibition of miR-25 ameliorates cardiac and skeletal muscle dysfunction in aged mdx/utrn haploinsufficient (+/−) mice

SV Kepreotis, JG Oh, M Park, J Yoo, C Lee… - … Therapy-Nucleic Acids, 2024 - cell.com
Dystrophic cardiomyopathy is a significant feature of Duchenne muscular dystrophy (DMD).
Increased cardiomyocyte cytosolic calcium (Ca 2+) and interstitial fibrosis are major …