Results 51 to 60 of about 86,962 (305)

Interleukin‐6 as a Key Biomarker in Facioscapulohumeral Dystrophy: Evidence From Longitudinal Analyses

Annals of Clinical and Translational Neurology, EarlyView.
ABSTRACT Objective Facioscapulohumeral muscular dystrophy type 1 (FSHD1) is a progressive neuromuscular disorder with no approved treatments. Identifying reliable biomarkers is critical to monitor disease severity, activity, and progression. Interleukin‐6 (IL‐6) has been proposed as a candidate biomarker, but longitudinal validation is limited ...
Jonathan Pini, Emanuela Martinuzzi, Sandra Dhifallah, Abderhmane Slioui, Angela Puma, Luisa Villa, Michele Cavalli, Andra Ezaru, Jérémy Garcia, Manuela Gambella, Federico Torre, Luca Jacopo Pavan, Nicolas Glaichenhaus, Sabrina Sacconi   +13 more
wiley   +1 more source

Nintedanib decreases muscle fibrosis and improves muscle function in a murine model of dystrophinopathy [PDF]

, 2018
Duchenne muscle dystrophy (DMD) is a genetic disorder characterized by progressive skeletal muscle weakness. Dystrophin deficiency induces instability of the sarcolemma during muscle contraction that leads to muscle necrosis and replacement of muscle by ...
Díaz Manera, Jordi, Escudero Cuadrado, Luis María, Fernández Simón, Esther, Gallardo, Eduard, Gómez Gálvez, Pedro, Piñol Jurado, Patricia, Suárez Calvet, Xavier   +6 more
core   +1 more source

Versatile Cell Penetrating Peptide for Multimodal CRISPR Gene Editing in Primary Stem Cells

Advanced Functional Materials, EarlyView.
CRISPR machinery in diverse molecular formats (DNA, RNA, and ribonucleic protein) is complexed into nanoparticles with the cell‐friendly arginine‐alanine‐leucine‐alanine (RALA) cell‐penetrating peptide. Nanoparticles are delivered to primary mesenchymal stem cells ex vivo or locally in vivo to facilitate multimodal CRISPR gene editing. This RALA‐CRISPR
Joshua P. Graham, Jose Gabriel Castro, Lisette C. Werba, Joseph Amitrano, Luke Fardone, Kevin P. Francis, Anand Ramamurthi, Michael J. Layden, Helen O. McCarthy, Tomas Gonzalez‐Fernandez   +9 more
wiley   +1 more source

Gene editing restores dystrophin expression in a canine model of Duchenne muscular dystrophy [PDF]

, 2018
Mutations in the gene encoding dystrophin, a protein that maintains muscle integrity and function, cause Duchenne muscular dystrophy (DMD). The deltaE50-MD dog model of DMD harbors a mutation corresponding to a mutational “hotspot” in the human DMD gene.
Amoasii, L, Bassel-Duby, R, Caballero, D, Harron, R, Hildyard, J C W, Li, H, Massey, C A, Mireault, A, Olson, E N, Piercy, R J, Sanchez-Ortiz, E, Shelton, J M, Stathopoulou, T-R   +12 more
core   +2 more sources

Anticodon Engineered Transfer RNA (tRNASUAG) Inhibits Hepatitis B Virus Replication by Promoting the Degradation of Core Protein

Advanced Science, EarlyView.
This study employed tRNASUAG, an anticodon‐engineered tRNA (ACE‐tRNA), to introduce a phosphorylatable serine into the C‐terminal domain of HBc by reading through its stop codon, thereby potentially promoting the ubiquitin‐proteasome degradation of HBc, and subsequently inhibiting HBV replication. Through a tRNASUAG‐gHBV1‐tRNASUAG‐gHBV2‐tRNASUAG tandem
Xingwen Yang, Huiying Sun, Ziheng Luo, Qinxin Zhang, Yarong Song, Jie Li, Xiaoyun Liu, Jie Wang   +7 more
wiley   +1 more source

Epidemiology and healthcare resource utilisation associated with Duchenne muscular dystrophy

Journal of Rare Diseases
Background This study aimed to describe the prevalence of Duchenne muscular dystrophy (DMD) and associated healthcare resource utilisation and costs from an England perspective.
Christopher Llewellyn Morgan, Josie Godfrey, Fleur Chandler, Emily Reuben, Craig J. Currie   +4 more
doaj   +1 more source

Functional characterization of orbicularis oculi and extraocular muscles [PDF]

, 2016
The orbicularis oculi are the sphincter muscles of the eyelids and are involved in modulating facial expression. They differ from both limb and extraocular muscles (EOMs) in their histology and biochemistry. Weakness of the orbicularis oculi muscles is a
Goldblum, David, Palmowski-Wolfe, Anja, Sekulic-Jablanovic, Marijana, Treves, Susan, Ullrich, Nina D., Zorzato, Francesco   +5 more
core   +2 more sources

Targeting PKCθ promotes satellite cell self-renewal [PDF]

, 2020
Skeletal muscle regeneration following injury depends on the ability of satellite cells (SCs) to proliferate, self-renew, and eventually differentiate. The factors that regulate the process of self-renewal are poorly understood. In this study we examined
Benedetti, Anna, Bouché, Marina, Fiore, Piera Filomena, Lozanoska-Ochser, Biliana, Madaro, Luca   +4 more
core   +1 more source

TREM2‐Mediated Cholesterol Efflux in Macrophages Inhibits Anti‐Tumor Immunity via Limitation of CD4+ T and NK Cells

Advanced Science, EarlyView.
Impaired anti‐tumor immunity is mediated by TREM2 + macrophages in non‐small cell lung cancer. TREM2 + macrophages prevent anti‐tumor immunity depending on the limitation of CD4 + T and NK cells. TREM2 promotes cholesterol efflux via ABCA1 to limit the production of CX3CL1 in macrophages.
Yunhan Wang, Weina Yu, Xinxin Wang, Qitai Zhao, Qingyang Lei, Aitian Li, Shasha Liu, Tian Wang, Li Yang, Yi Zhang   +9 more
wiley   +1 more source

Distrofina na diferenciação das distrofias de duchenne e becker estudo imuno-histoquímico comparado com o estádio clínico, enzimas séricas e biópsia muscular

Arquivos de Neuro-Psiquiatria, 1990
Foram estudados 55 casos de distrofia muscular progressiva (34 Duchenne, 12 Duchenne com distrofina residual e 9 Becker), comparando idade, época de início e tempo de sintomas, graduação na escala de Vignos e Archibald, níveis de enzimas séricas e ...
Lineu Cesar Werneck, Eduardo Bonilla
doaj   +1 more source