Clinical advances of RNA therapeutics for treatment of neurological and neuromuscular diseases

A Holm, SN Hansen, H Klitgaard, S Kauppinen - RNA biology, 2022 - Taylor & Francis
ABSTRACT RNA therapeutics comprise a diverse group of oligonucleotide-based drugs
such as antisense oligonucleotides (ASOs), small interfering RNAs (siRNAs), and short …

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 …

The effect of uridine on the state of skeletal muscles and the functioning of mitochondria in Duchenne dystrophy

MV Dubinin, VS Starinets, NV Belosludtseva… - International Journal of …, 2022 - mdpi.com
Duchenne muscular dystrophy is caused by the loss of functional dystrophin that secondarily
causes systemic metabolic impairment in skeletal muscles and cardiomyocytes. The …

Sarcoplasmic Reticulum Ca2+ Buffer Proteins: A Focus on the Yet-To-Be-Explored Role of Sarcalumenin in Skeletal Muscle Health and Disease

E Conte, G Dinoi, P Imbrici, A De Luca, A Liantonio - Cells, 2023 - mdpi.com
Sarcalumenin (SAR) is a luminal Ca2+ buffer protein with high capacity but low affinity for
calcium binding found predominantly in the longitudinal sarcoplasmic reticulum (SR) of fast …

BKCa Activator NS1619 Improves the Structure and Function of Skeletal Muscle Mitochondria in Duchenne Dystrophy

MV Dubinin, VS Starinets, NV Belosludtseva… - Pharmaceutics, 2022 - mdpi.com
Duchenne muscular dystrophy (DMD) is a progressive hereditary disease caused by the
absence of the dystrophin protein. This is secondarily accompanied by a dysregulation of …

Therapeutic Application of Extracellular Vesicles-Capsulated Adeno-Associated Virus Vector via nSMase2/Smpd3, Satellite, and Immune Cells in Duchenne …

Y Matsuzaka, Y Hirai, K Hashido, T Okada - International Journal of …, 2022 - mdpi.com
Duchenne muscular dystrophy (DMD) is caused by loss-of-function mutations in the
dystrophin gene on chromosome Xp21. Disruption of the dystrophin–glycoprotein complex …

Mineralocorticoid receptor signaling in the inflammatory skeletal muscle microenvironments of muscular dystrophy and acute injury

ZM Howard, CK Gomatam, AB Piepho… - Frontiers in …, 2022 - frontiersin.org
Duchenne muscular dystrophy (DMD) is a striated muscle degenerative disease due to loss
of functional dystrophin protein. Loss of dystrophin results in susceptibility of muscle …

Effect of Large-Conductance Calcium-Dependent K+ Channel Activator NS1619 on Function of Mitochondria in the Heart of Dystrophin-Deficient Mice

MV Dubinin, VS Starinets, YA Chelyadnikova… - Biochemistry …, 2023 - Springer
Dystrophin-deficient muscular dystrophy (Duchenne dystrophy) is characterized by impaired
ion homeostasis, in which mitochondria play an important role. In the present work, using a …

Does β-Hydroxy-β-Methylbutyrate Have Any Potential to Support the Treatment of Duchenne Muscular Dystrophy in Humans and Animals?

AE Gorji, P Ostaszewski, K Urbańska, T Sadkowski - Biomedicines, 2023 - mdpi.com
Skeletal muscle is the protein reservoir of our body and an important regulator of glucose
and lipid homeostasis. The dystrophin gene is the largest gene and has a key role in …

Hypochlorous acid exposure impairs skeletal muscle function and Ca2+ signalling: implications for Duchenne muscular dystrophy pathology

TA Lea, PM Panizza, PG Arthur… - The Journal of …, 2023 - Wiley Online Library
Duchenne muscular dystrophy (DMD) is a fatal X‐linked disease characterised by severe
muscle wasting. The mechanisms underlying the DMD pathology likely involve the …