Results 51 to 60 of about 71,938 (313)

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

Noninvasive ventilatory support to reverse weight loss in Duchenne muscular dystrophy: A case series

Pulmonology, 2019
This case series of five patients with Duchenne muscular dystrophy demonstrates the nutritional advantages of instituting noninvasive intermittent positive pressure ventilatory support via 15 mm angled mouthpieces to relieve tachypnea and provide more ...
P. Deo, J.R. Bach
doaj   +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

Payload‐Driven Design of Polymeric Carriers for Nucleic Acid Delivery: Insights from Structure–Function Relationships

Advanced Science, EarlyView.
Polymeric carriers provide versatile platforms for nucleic acid delivery. This review introduces a novel perspective inspired by protein structural organization to explore structure–function relationships of polymeric carriers, highlighting requirements of different nucleic acid payloads.
Hanieh Moradian, Charlotte Maeve Dunne, Manfred Gossen, Matthias Hartlieb   +3 more
wiley   +1 more source

Feasibility and tolerability of whole-body, low-intensity vibration and its effects on muscle function and bone in patients with dystrophinopathies: a pilot study. [PDF]

, 2017
IntroductionDystrophinopathies are X-linked muscle degenerative disorders that result in progressive muscle weakness complicated by bone loss. This study's goal was to evaluate feasibility and tolerability of whole-body, low-intensity vibration (WBLIV ...
Grames, Molly, Hodges, James S, Karachunski, Peter, Lowe, Dawn A, Petryk, Anna, Polgreen, Lynda E   +5 more
core   +1 more source

The Evolutionarily Conserved TPM1 Super‐Enhancer Drives Skeletal Muscle Regeneration via Mechanotransduction Signaling

Advanced Science, EarlyView.
By integrating biomechanical and epigenetic cues, the evolutionarily conserved TPM1 super‐enhancer drives myogenesis via TEAD4‐mediated chromatin looping. This mechanism produces species‐specific outputs (linear TPM1 mRNA in mice and CircTPM1 in bovine) that activate PI3K/AKT mechanotransduction and the MYH10/MYL3 axis to execute cytoskeletal ...
Ruimen Zhang, Wanyou Feng, Yanyan Yang, Yu Pan, Chaoxia Zou, Leyi Wang, Sanbao Zhang, Yimin Zhao, Yongmei Wu, Jinling Wang, Jianwei Zou, Kehe Cen, Yongwang Zhang, Han Huang, Yurong Xu, Li Zhong, Hailong Gong, Juanru Cheng, Jingyuan Liang, Zihua Zheng, Qinyang Jiang, Jingwei Wei, Hui Li, Minhui Liang, Deshun Shi, Sufang Yang, Yanfei Deng, Yingming Wei   +27 more
wiley   +1 more source

Co‐Opting MBNL‐Dependent Alternative Splicing Cassette Exons to Control Gene Therapy in Myotonic Dystrophy

Annals of Neurology, EarlyView.
Objective Myotonic dystrophy type 1 (DM1) is a highly variable, multisystemic genetic disorder caused by a CTG repeat expansion in the 3′ untranslated region of DMPK. Toxicity is exerted by repeat‐containing DMPK transcripts that sequester muscleblind‐like (MBNL) proteins and lead to deleterious yet predictable changes in alternative splicing.
Samuel T. Carrell, Ellie M. Carrell, Ryan Giovenco, Beverly L. Davidson   +3 more
wiley   +1 more source

Preimplantation genetic diagnosis associated to Duchenne muscular dystrophy

Einstein (São Paulo)
Duchenne muscular dystrophy is the most common muscle disease found in male children. Currently, there is no effective therapy available for Duchenne muscular dystrophy patients. Therefore, it is essential to make a prenatal diagnosis and provide genetic
Bianca Bianco, Denise Maria Christofolini, Gabriel Seixas Conceição, Caio Parente Barbosa   +3 more
doaj   +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

A Novel Transcriptional Slippage Mechanism Rescues Dystrophin Expression from a DMD Frameshift Variant

Annals of Neurology, EarlyView.
Pathogenic DMD variants usually follow the reading‐frame rule: out‐of‐frame changes cause Duchenne muscular dystrophy, whereas in‐frame ones produce Becker muscular dystrophy (BMD). We report a 23‐year‐old man with BMD‐like weakness, calf hypertrophy, elevated creatine kinase, and dilated cardiomyopathy.
Hiroya Naruse, Jun Mitsui, Akatsuki Kubota, So Okubo, Shuichiro Mitsuchi, Kensho Sumi, Shuichi Tanifuji, Shogo Komaki, Asuka Kitamura, Meiko Maeda, Daiki Yashita, Atsushi Sudo, Takashi Matsukawa, Masashi Hamada, Wataru Satake, Shoji Tsuji, Tatsushi Toda   +16 more
wiley   +1 more source