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 …
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 …
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 …
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 …
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
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 …
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 …
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 …
dystrophin, a protein essential for myocyte integrity. Mitochondrial dysfunction is reportedly …
GDF8 inhibition enhances musculoskeletal recovery and mitigates posttraumatic osteoarthritis following joint injury
Musculoskeletal disorders contribute substantially to worldwide disability. Anterior cruciate
ligament (ACL) tears result in unresolved muscle weakness and posttraumatic osteoarthritis …
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 …
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 …
as a potential therapeutic target in muscular dystrophies and other conditions. Preclinical …