Engineered RNA Devices for In Vivo Targeted Therapeutics via Advanced Delivery Systems
Aggregate, EarlyView.Schematic illustration of engineered RNA devices for in vivo targeted therapeutics via advanced delivery systems. ABSTRACT Engineered RNA devices can identify disease‐specific markers and precisely regulate gene expression, which is of great significance to the development of precision medicine.
Wei Luo +6 more
wiley +1 more source
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 +3 more
wiley +1 more source
A Roadmap to Newborn Screening for Duchenne Muscular Dystrophy
International Journal of Neonatal Screening, 2017 Duchenne muscular dystrophy (DMD) is the most common childhood form of muscular dystrophy, with an estimated frequency of 1:5000 live births. The impact of the disease presents as early as infancy with significant developmental delays, and ultimately ...
Samiah A. Al-Zaidy +4 more
doaj +1 more source
Modulation of PGC-1α activity as a treatment for metabolic and muscle-related diseases [PDF]
, 2014 Physical inactivity is a predisposing factor for various disease states including obesity, cardiovascular disease, as well as for certain types of cancer. Regular endurance exercise mediates several beneficial effects such as increased energy expenditure
Handschin, Christoph +1 more
core +1 more source
Nerve damage induced skeletal muscle atrophy is associated with increased accumulation of intramuscular glucose and polyol pathway intermediates [PDF]
, 2020 Perturbations in skeletal muscle metabolism have been reported for a variety of neuromuscular diseases. However, the role of metabolism after constriction injury to a nerve and the associated muscle atrophy is unclear.
Afzal, Shoaib +3 more
core +2 more sources
Journal of NeuroEngineering and Rehabilitation, 2012 Background Compensatory trunk movements during gait, such as a Duchenne limp, are observed frequently in subjects with osteoarthritis of the hip, yet angular trunk movements are seldom included in clinical gait assessments.
Reininga Inge HF +5 more
doaj +1 more source
Frontiers in Cell and Developmental Biology, 2022 In vitro models of patient-derived muscle allow for more efficient development of genetic medicines for the muscular dystrophies, which often present mutation-specific pathologies.
Florian Barthélémy +14 more
doaj +1 more source
Contrasting Experimentally Device-Manipulated and Device-Free Smiles. [PDF]
, 2019 Researchers in psychology have long been interested in not only studying smiles, but in examining the downstream effects of experimentally manipulated smiles.
Cross, Marie P +2 more
core
Multicolor fluorescence in situ hybridization on metaphase chromosomes and interphase Halo-preparations using cosmid and YAC clones for the simultaneous high resolution mapping of deletions in the dystrophin gene [PDF]
, 1994 We report on multicolor fluorescence in situ hybridization protocols for the simultaneous visualization of deletion-prone regions for carrier detection of Duchenne/ Becker (DMD/BMD) muscular dystrophy.
Blonden, Lau +6 more
core +1 more source
A Psychological Guide to Upper Face Botulinum Toxin Injections: Baseline Emotional Functions of Facial Expressions. [PDF]
J Cosmet DermatolABSTRACT Background The upper face is central to human nonverbal communication, with the glabellar complex, forehead, and lateral canthal area signaling core emotions such as anger, sadness, fear, surprise, and joy. Botulinum toxin type A (BoNT‐A) is widely used to modulate muscle activity in these regions, not only reducing dynamic wrinkles but also ...
Hopf AGM +4 more
europepmc +2 more sources