A functional motor unit in the culture dish : co-culture of spinal cord explants and muscle cells [PDF]
, 2012 Human primary muscle cells cultured aneurally in monolayer rarely contract spontaneously because, in the absence of a nerve component, cell differentiation is limited and motor neuron stimulation is missing(1). These limitations hamper the in vitro study
Arnold, Anne-Sophie +2 more
core +1 more source
The burden, epidemiology, costs and treatment for Duchenne muscular dystrophy: an evidence review
Orphanet Journal of Rare Diseases, 2017 BackgroundDuchenne Muscular Dystrophy (DMD) is a rapidly progressive, lethal neuromuscular disorder, present from birth, which occurs almost exclusively in males. We have reviewed contemporary evidence of burden, epidemiology, illness costs and treatment
S. Ryder +7 more
semanticscholar +1 more source
Nature Inspired Delivery Vehicles for CRISPR‐Based Genome Editing
Small, EarlyView.The review highlights nature‐inspired nanocarriers for CRISPR delivery, emphasizing viral vectors, extracellular vesicles, liposomes, and lipid nanoparticles. It discusses their roles in improving specificity, minimizing immunogenicity, and overcoming barriers in genome editing. Recent advancements, challenges, and therapeutic applications are explored,
Elizabeth Maria Clarissa +4 more
wiley +1 more source
Let the consumer decide? The regulation of commercial genetic testing. [PDF]
, 2001 Objectives—The development of predictive genetic tests provides a new area where consumers can gain knowledge of their health status and commercial opportunities.
Levitt, M. I.
core +3 more sources
Reviewer #1 (Public Review): Development and validation of a high throughput screening platform to enable target identification in skeletal muscle cells from Duchenne Muscular Dystrophy (DMD) patients [PDF]
, 2023 Santosh Hariharan +12 more
openalex +1 more source
Lipid Nanoparticles for Delivery of CRISPR Gene Editing Components
Small Methods, EarlyView.The review presents a comprehensive overview of each component of lipid nanoparticles（LNPs）and their effects on editing efficiency. It specifically highlights strategies for achieving non‐liver delivery, aiming for broader applications in gene editing. Furthermore, this review summarizes the applications of LNPs in gene editing and offers insights for ...
Fan Wu +6 more
wiley +1 more source
Induced Pluripotent Stem Cells for Duchenne Muscular Dystrophy Modeling and Therapy
Cells, 2018 Duchenne muscular dystrophy (DMD) is an X-linked recessive disorder, caused by mutation of the DMD gene which encodes the protein dystrophin. This dystrophin defect leads to the progressive degeneration of skeletal and cardiac muscles.
Lubos Danisovic +2 more
doaj +1 more source
Prenatal genetic counselors' attitudes, beliefs, and practices with discussing postnatal Duchenne muscular dystrophy treatment options. [PDF]
J Genet CounsAbstract Duchenne muscular dystrophy (DMD) is an X‐linked genetic condition characterized by progressive muscle degeneration. Recently, several genotype‐specific treatment options for DMD have become available. Prenatal genetic counselors may encounter at‐risk patients for DMD, through a variety of indications.
Jurlina SL +3 more
europepmc +2 more sources
World Journal of Otorhinolaryngology - Head and Neck Surgery, EarlyView.ABSTRACT Spasmodic dysphonia is a laryngeal dystonia that can present as adductor, abductor, or mixed types, with or without tremor. The etiology is not understood fully. Comprehensive evaluation is required to establish the diagnosis. Treatments include voice therapy, medications, botulinum toxin injection, laryngeal surgery, deep brain stimulation ...
Aaron J. Jaworek, Robert T. Sataloff
wiley +1 more source
The importance of genetic diagnosis for Duchenne muscular dystrophy
Journal of Medical Genetics, 2016 Duchenne muscular dystrophy (DMD) and Becker muscular dystrophy are caused by mutations in the dystrophin-encoding DMD gene. Large deletions and duplications are most common, but small mutations have been found as well.
Annemieke Aartsma-Rus +2 more
semanticscholar +1 more source