Results 61 to 70 of about 56,165 (274)

Genetic Code Expanded T Cell for Controllable Immunotherapy

Advanced Science, EarlyView.
Our GCE‐CAR‐T cells enables tight, dose‐dependent, and function‐preserving control of CAR expression at the translational level through amber codon suppression and genetic incorporation of ncAA. ABSTRACT Chimeric antigen receptor (CAR)‐T cell therapy has demonstrated curative potential against hematologic malignancies, but its clinical application ...
Xue Wang, Yingao Gao, Yeyu Su, Yong Wang, Tao Liu   +4 more
wiley   +1 more source

Impaired regeneration of dystrophin-deficient muscle fibers is caused by exhaustion of myogenic cells

Brazilian Journal of Medical and Biological Research, 2002
Duchenne muscular dystrophy is one of the most devastating myopathies. Muscle fibers undergo necrosis and lose their ability to regenerate, and this may be related to increased interstitial fibrosis or the exhaustion of satellite cells. In this study, we
M.A.M. Luz, M.J. Marques, H. Santo Neto
doaj   +1 more source

The Role of Extracellular Vesicles MicroRNAs in Sarcopenia: From Aging to Multi‐Morbidity

AGING MEDICINE, EarlyView.
Exosomes transporting miRNAs play a crucial bidirectional regulatory role in the process of sarcopenia, both in natural aging and under various pathological conditions. ABSTRACT Sarcopenia, defined as progressive loss of skeletal muscle mass and function, occurs during aging and has also been recognized for its detrimental effects in various disease ...
Bingyu Huang, Zhao Peng, Lin Kang
wiley   +1 more source

Swallowing and Communication in Cockayne Syndrome: Clinical Characteristics and Management

American Journal of Medical Genetics Part A, EarlyView.
ABSTRACT Cockayne syndrome (CS) is an ultrarare genetic disorder associated with genes encoding proteins involved in DNA repair. The clinical course of CS involves neurodevelopmental and neurodegenerative features, including swallowing and communication impairments.
Abigail M. Spoden, Katlyn E. McGrattan, Peter B. Kang   +2 more
wiley   +1 more source

The golden retriever model of Duchenne muscular dystrophy

Skeletal Muscle, 2017
Duchenne muscular dystrophy (DMD) is an X-linked disease caused by mutations in the DMD gene and loss of the protein dystrophin. The absence of dystrophin leads to myofiber membrane fragility and necrosis, with eventual muscle atrophy and contractures ...
Joe N. Kornegay
doaj   +1 more source

Metformin enhances external urethral sphincter integrity and restores continence via AMPK activation in a rat model of stress urinary incontinence

Animal Models and Experimental Medicine, EarlyView.
The pathogenesis of stress urinary incontinence (SUI) is intimately associated with injury to the external urethral sphincter (EUS). In this study, we established an SUI model induced by double vaginal distension and demonstrated that metformin treatment activated the AMPK signaling in the EUS tissue.
Yuting Xu, Xuhong Li, Alvaro Munoz, Li Jiang, Lihua Huang, Yanhua Zhou, Liping Zhu, Youbo Yang   +7 more
wiley   +1 more source

Direct myocardial implantation of human embryonic stem cells in a dog model of Duchenne cardiomyopathy reveals poor cell survival in dystrophic tissue

Journal of Stem Cells and Regenerative Medicine, 2011
Duchenne muscular dystrophy is characterized by progressive muscle weakness and early death resulting from dystrophin deficiency. Spontaneous canine muscular disorders are interesting settings to evaluate the relevance of innovative therapies in human ...
Habeler W, Ghaleh B, Barthelemy I, Plancheron A, Bizé A, Carlos-Sampedrano C, Chetboul V, Berdeaux A, Pucéat M, Peschanski M, Blot S, Monville C   +11 more
doaj  

The UK myotonic dystrophy patient registry: facilitating and accelerating clinical research [PDF]

, 2017
Myotonic dystrophy type 1 (DM1) is the most frequent muscular dystrophy worldwide with complex, multi-systemic, and progressively worsening symptoms. There is currently no treatment for this inherited disorder and research can be challenging due to the ...
Atalaia, Antonio, Cordts, Isabell, Hammans, Simon, Hilton-Jones, David, Jimenez-Moreno, Aura Cecilia, Lochmüller, Hanns, Maddison, Paul, Marini-Bettolo, Chiara, Monckton, Darren G., Nikolenko, Nikoletta, Orrell, Richard, Petty, Richard, Phillips, Margaret, Roberts, Mark, Rogers, Mark, Straub, Volker, Thompson, Rachel, Turner, Chris, Wood, Libby   +18 more
core   +4 more sources

Evaluation of quantitative muscle MRI and an intelligent phenotyping housing system as advanced phenotyping methods in a mouse model of calpain 3‐deficient muscular dystrophy

Animal Models and Experimental Medicine, EarlyView.
We applied quantitative MRI of the lower limb and automated home‐cage phenotyping to a mouse model of calpainopathy to detect early disease changes. At 15 months, calpain 3‐deficient mice showed increased water T2 values correlating with immune cell infiltration in the soleus and gastrocnemius muscles, while assessment of motor activity revealed only ...
Nicolina Südkamp, Marlena Rohm, Gabriele Russo, Xavier Helluy, Abdulhadi Kocabas, Martijn Froeling, Felix Kleefeld, Denise Manahan‐Vaughan, Tobias Ruck, Frank Jacobsen, Matthias Vorgerd, Johannes Forsting, Lara Schlaffke   +12 more
wiley   +1 more source

Cycles of myofiber degeneration and regeneration lead to remodeling of the neuromuscular junction in two mammalian models of Duchenne muscular dystrophy.

PLoS ONE, 2018
Mice lacking the sarcolemmal protein dystrophin, designated mdx, have been widely used as a model of Duchenne muscular dystrophy. Dystrophic mdx mice as they mature develop notable morphological abnormalities to their neuromuscular junctions, the ...
Seth G Haddix, Young Il Lee, Joe N Kornegay, Wesley J Thompson   +3 more
doaj   +1 more source