Results 51 to 60 of about 55,853 (286)

Evaluation of 2’-Deoxy-2’-fluoro Antisense Oligonucleotides for Exon Skipping in Duchenne Muscular Dystrophy

open access: yesMolecular Therapy: Nucleic Acids, 2015
Duchenne muscular dystrophy (DMD) is a severe muscle wasting disorder typically caused by frame-shifting mutations in the DMD gene. Restoration of the reading frame would allow the production of a shorter but partly functional dystrophin protein as seen ...
Silvana M G Jirka   +7 more
doaj   +1 more source

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

open access: yes, 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   +18 more
core   +4 more sources

Nanomaterial‐Based Muscle Cell/Neural Tissue Biohybrid Robots: From Actuation to Biomedical Applications

open access: yesAdvanced Robotics Research, EarlyView.
Muscle cell‐based biohybrid robot using nanomaterials for function enhancement and neural function for biomedical applications. Biohybrid robotics, an emerging field combining biological tissues with artificial systems, has made significant progress in developing various biohybrid constructs, including muscle‐cell‐driven biorobots and microbots.
Minkyu Shin   +4 more
wiley   +1 more source

Human Adipose-Derived Mesenchymal Stromal Cells Injected Systemically into GRMD Dogs without Immunosuppression are Able to Reach the Host Muscle and Express Human Dystrophin

open access: yesCell Transplantation, 2012
Duchenne muscular dystrophy (DMD), a lethal X-linked disorder, is the most common and severe form of muscular dystrophies, affecting 1 in 3,500 male births. Mutations in the DMD gene lead to the absence of muscle dystrophin and a progressive degeneration
N. M. Vieira   +12 more
doaj   +1 more source

Immunity and AAV-mediated gene therapy for muscular dystrophies in large animal models and human trials

open access: yesFrontiers in Microbiology, 2011
Adeno-associated viral (AAV) vector mediated gene replacement for the treatment of muscular dystrophy represents a promising therapeutic strategy in modern medicine.
Zejing eWang   +6 more
doaj   +1 more source

Altered splicing of the BIN1 muscle-specific exon in humans and dogs with highly progressive centronuclear myopathy [PDF]

open access: yes, 2013
Amphiphysin 2, encoded by BIN1, is a key factor for membrane sensing and remodelling in different cell types. Homozygous BIN1 mutations in ubiquitously expressed exons are associated with autosomal recessive centronuclear myopathy (CNM), a mildly ...
A Buj-Bello   +58 more
core   +7 more sources

Calcium Shock Enables Efficient and Programmable Particle Delivery for Genome Editing Applications

open access: yesAdvanced Science, EarlyView.
Classical transfection and transduction are inefficient, particularly with confluent cells and organoids, and lack cell type‐specific programmability. This study presents calcium shock (CaSh), a method that dramatically improves particle delivery into single cells, colonies, and organoids.
Nicole Vo   +12 more
wiley   +1 more source

Utrophin, MHC and M1/M2 macrophages in GRMD dogs

open access: yesCiência Animal Brasileira, 2020
Muscular dystrophies are hereditary diseases that lead to progressive degeneration of the skeletal musculature. Golden Retriever dogs are used as animal models because they show a hereditary muscle disease similar to muscular dystrophy in humans.
Gabriela Noronha de Toledo   +1 more
doaj  

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

open access: yesBrazilian 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

LMO7 Suppresses Tumor‐Associated Macrophage Phagocytosis of Tumor Cells Through Degradation of LRP1

open access: yesAdvanced Science, EarlyView.
LMO7 in tumor‐associated macrophages suppresses phagocytosis of tumor cells and limits cytotoxic T lymphocytes infiltration, fostering tumor progression. Mechanistically, LMO7 mediates the ubiquitination and degradation of the phagocytic receptor LRP1, impairing its ability to engulf tumor cells and driving macrophages toward an antitumor phenotype ...
Mengkai Li   +12 more
wiley   +1 more source

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