CRISPR-based therapeutic gene editing for Duchenne muscular dystrophy: advances, challenges and perspectives

G Chen, T Wei, H Yang, G Li, H Li - Cells, 2022 - mdpi.com
Duchenne muscular dystrophy (DMD) is a severe neuromuscular disease arising from loss-
of-function mutations in the dystrophin gene and characterized by progressive muscle …

GDF15 neutralization restores muscle function and physical performance in a mouse model of cancer cachexia

JY Kim-Muller, LJ Song, BLC Paulhus, E Pashos, X Li… - Cell Reports, 2023 - cell.com
Cancer cachexia is a disorder characterized by involuntary weight loss and impaired
physical performance. Decline in physical performance of patients with cachexia is …

Current pharmacological strategies for Duchenne muscular dystrophy

S Yao, Z Chen, Y Yu, N Zhang, H Jiang… - Frontiers in Cell and …, 2021 - frontiersin.org
Duchenne muscular dystrophy (DMD) is a lethal, X-linked neuromuscular disorder caused
by the absence of dystrophin protein, which is essential for muscle fiber integrity. Loss of …

The D2.mdx mouse as a preclinical model of the skeletal muscle pathology associated with Duchenne muscular dystrophy

DW Hammers, CC Hart, MK Matheny, LA Wright… - Scientific reports, 2020 - nature.com
Duchenne muscular dystrophy (DMD) is an X-linked, lethal muscle degenerative disease
caused by loss of dystrophin protein. DMD has no cure and few treatment options …

The failed clinical story of myostatin inhibitors against Duchenne muscular dystrophy: exploring the biology behind the battle

E Rybalka, CA Timpani, DA Debruin, RM Bagaric… - Cells, 2020 - mdpi.com
Myostatin inhibition therapy has held much promise for the treatment of muscle wasting
disorders. This is particularly true for the fatal myopathy, Duchenne Muscular Dystrophy …

[HTML][HTML] Randomized phase 2 trial and open-label extension of domagrozumab in Duchenne muscular dystrophy

KR Wagner, HZ Abdel-Hamid, JK Mah… - Neuromuscular …, 2020 - Elsevier
We report results from a phase 2, randomized, double-blind, 2-period trial (48 weeks each)
of domagrozumab and its open-label extension in patients with Duchenne muscular …

The effect of deflazacort treatment on the functioning of skeletal muscle mitochondria in duchenne muscular dystrophy

MV Dubinin, EY Talanov, KS Tenkov… - International journal of …, 2020 - mdpi.com
Duchenne muscular dystrophy (DMD) is a severe hereditary disease caused by a lack of
dystrophin, a protein essential for myocyte integrity. Mitochondrial dysfunction is reportedly …

GDF8 inhibition enhances musculoskeletal recovery and mitigates posttraumatic osteoarthritis following joint injury

CR Brightwell, CM Latham, AR Keeble, NT Thomas… - Science …, 2023 - science.org
Musculoskeletal disorders contribute substantially to worldwide disability. Anterior cruciate
ligament (ACL) tears result in unresolved muscle weakness and posttraumatic osteoarthritis …

[HTML][HTML] The elusive promise of myostatin inhibition for muscular dystrophy

KR Wagner - Current opinion in neurology, 2020 - journals.lww.com
The elusive promise of myostatin inhibition for muscular dys... : Current Opinion in Neurology
The elusive promise of myostatin inhibition for muscular dystrophy : Current Opinion in …

Antimyostatin treatment in health and disease: The story of great expectations and limited success

TL Nielsen, J Vissing, TO Krag - Cells, 2021 - mdpi.com
In the past 20 years, myostatin, a negative regulator of muscle mass, has attracted attention
as a potential therapeutic target in muscular dystrophies and other conditions. Preclinical …