Current Muscular Dystrophy News and Events

Current Muscular Dystrophy News and Events, Muscular Dystrophy News Articles.
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New method identifies adaptive mutations in complex evolving populations
A research team co-led by a scientist at the Hong Kong University of Science and Technology has developed a method to study how HIV mutates to escape the immune system in multiple patients, which could inform HIV vaccine design. (2020-11-30)

Genetic treatment plus exercise reverses fatigue in mice with muscle wasting disease
Adding exercise to a genetic treatment for myotonic dystrophy type 1 was more effective at reversing fatigue than administering the treatment alone in a study using a mouse model of the disease. In fact, exercise alone provided some benefit whereas the genetic treatment alone did not. This study, carried out by researchers at Massachusetts General Hospital and collaborators, has implications for patients with fatigue due to genetics-related musculoskeletal diseases and other types of illness-induced fatigue. (2020-11-30)

Men tuning into Insta-spiration
New research confirms men are affected by Instagram influencers who set global benchmarks for ideal body shape, fashion and even facial trends. While perhaps not as focused on 'thinness' as women appear to be from female influencers, the Flinders University study confirms males are responding to the body image and fitness messages shared by Instagram leaders, some with millions of followers. (2020-11-29)

Trinity researchers discover how the brain 're-wires' after disease
Trinity College researchers are studying how the brain re-wires itself in neurological disease. The team is building treatments for today's more common global conditions like Motor Neurone Disease (MND/ALS) and Spinal Muscular Atrophy and their findings could impact rehabilitation for patients, the discovery of effective drugs and quantifying the potential efficacy of new therapies. (2020-11-24)

Mother's touch lingers in her child's genes
Mothers leave their mark on their children in many ways - and Melbourne researchers have discovered a protein called SMCHD1 is involved in this 'imprinting' process. SMCHD1 switches certain genes off, altering how a cell behaves. The new research has revealed that when an egg cell (or oocyte) is fertilised by a sperm, the egg cell's SMCHD1 lingers within the developing embryo, switching off at least 10 different genes and impacting the embryo's development - which could potentially have a lifelong impact on the offspring. (2020-11-23)

Revolutionary CRISPR-based genome editing system treatment destroys cancer cells
Researchers at Tel Aviv University (TAU) have demonstrated that the CRISPR/Cas9 system is very effective in treating metastatic cancers, a significant step on the way to finding a cure for cancer. The researchers developed a novel lipid nanoparticle-based delivery system that specifically targets cancer cells and destroys them by genetic manipulation. The system, called CRISPR-LNPs, carries a genetic messenger (messenger RNA), which encodes for the CRISPR enzyme Cas9 that acts as molecular scissors that cut the cells' DNA. (2020-11-18)

Penn researchers develop approach to prevent toxicity tied to neurological gene therapy
Penn Medicine researchers have developed a new targeted approach to prevent a toxicity seen in the sensory neurons of dorsal root ganglia after gene therapy to treat neurological disorders. It's an important hurdle to clear, as the field works toward more safe and effective gene therapies for patients with disorders like spinal muscular atrophy. (2020-11-11)

Low fitness linked to higher depression and anxiety risk
People with low aerobic and muscular fitness are nearly twice as likely to experience depression, finds a new study led by UCL researchers, published in BMC Medicine. (2020-11-10)

Two-birds-one-stone strategy shows promise in RNA-repeat expansion diseases
A new strategy for treating a variety of diseases known as RNA-repeat expansion disorders, which affect millions of people, has shown promise in proof-of-principle tests conducted by scientists at Scripps Research. (2020-11-06)

Genetic mutation could worsen heart function in Duchenne muscular dystrophy patients
DALLAS - Nov. 4, 2020 - A mutation in the gene that causes cystic fibrosis may accelerate heart function decline in those with Duchenne muscular dystrophy (DMD), a new study by UT Southwestern researchers suggests. The findings, published online recently in the Journal of the American Heart Association, could help doctors develop new strategies to preserve heart function in this population, potentially extending patients' lives. (2020-11-04)

Research lowers errors for using brain signals to control a robot arm
Brain-computer interfaces have seen a large influx of research in an effort to allow precise and accurate control of physical systems. By measuring brain signals and implementing a clever feedback scheme, researchers from India and the UK have reduced the positional error in brain-controlled robot arms by a factor of 10, paving the way to greatly enhancing the quality of life for people suffering from strokes and neuro-muscular disorders. (2020-10-28)

Brazilian researchers discover how muscle regenerates after exercise
Adaptation of muscle tissue to aerobic exercise alters the metabolism of muscle stem cells, helping them recover from injury. Findings may contribute to treatment of cachexia, sarcopenia and other conditions associated with lean mass loss. (2020-10-28)

COVID-19 anxiety linked to body image issues
A new study has found that anxiety and stress directly linked to COVID-19 could be causing a number of body image issues. The research, which involved 506 UK adults, found that worries linked to COVID-19 were associated with body dissatisfaction and a desire for thinness in women, and associated with body fat dissatisfaction and a desire for muscularity in men. (2020-10-22)

Scientists identify compound that stimulates muscle cells in mice
UCLA researchers have identified a compound that can reproduce the effect of exercise in muscle cells in mice. The findings are published in the journal Cell Reports Medicine. (2020-10-21)

Skeletal muscle development and regeneration mechanisms vary by gender
Researchers at Kumamoto University, Japan generated mice lacking the estrogen receptor beta (ERβ) gene, both fiber-specific and muscle stem cell-specific, which resulted in abnormalities in the growth and regeneration of skeletal muscle in female mice. This was not observed in male mice that lacked the ERβ gene, suggesting that estrogen and its downstream signals may be a female-specific mechanism for muscle growth and regeneration. (2020-10-12)

Damaged muscles don't just die, they regenerate themselves
Researchers building a model of muscle damage in a cultured system found that components leaking from broken muscle fibers activate ''satellite cells,'' which are muscle stem cells. While attempting to identify the activating proteins, they found that metabolic enzymes, such as GAPDH, rapidly activated quiescent satellite cells and accelerated muscle injury regeneration. This is a highly rational and efficient regeneration mechanism, in which the damaged muscle itself activates satellite cells for regeneration. (2020-10-12)

Terahertz zaps alter gene activity in stem cells
Terahertz light pulses change gene expression in stem cells, report researchers from Kyoto University's Institute for Integrated Cell-Material Sciences (iCeMS) and Tokai University in Japan in the journal Optics Letters. The findings come thanks to a new tool, with implications for stem cell research and regenerative therapy development. (2020-10-08)

Microneedles for therapeutic gene delivery
Researchers develop a minimally invasive biodegradable microneedle patch as a novel delivery mode for gene therapy applications (2020-10-07)

New Duchenne muscular dystrophy drug shows benefit in Duke trial
A new drug offers hope for young boys with the progressive neuromuscular disease Duchenne muscular dystrophy (DMD) by potentially offering an alternative to high-dose glucocorticoids that have significant side effects. (2020-09-25)

Scientists uncover a novel approach to treating Duchenne muscular dystrophy
Scientists at Sanford Burnham Prebys, Fondazione Santa Lucia IRCCS, and Università Cattolica del Sacro Cuore have shown that pharmacological (drug) correction of the content of extracellular vesicles released within dystrophic muscles can restore their ability to regenerate muscle and prevent muscle scarring. The study, published in EMBO Reports, reveals a promising new therapeutic approach for Duchenne muscular dystrophy (DMD), an incurable muscle-wasting condition, and has far-reaching implications for the field of regenerative medicine. (2020-09-15)

Twist on CRISPR gene editing treats adult-onset muscular dystrophy in mice
UC San Diego researchers demonstrate that one dose of their version of CRISR gene editing can chew up toxic RNA and almost completely reverse symptoms in a mouse model of myotonic dystrophy, a type of adult-onset muscular dystrophy. (2020-09-14)

Drugging the undruggable: Yale finds treatment path for muscular dystrophy
Researchers at Yale have identified a possible treatment for Duchenne muscular dystrophy (DMD), a rare genetic disease for which there is currently no cure or treatment, by targeting an enzyme that had been considered ''undruggable.'' The finding appears in the Aug. 25 edition of Science Signaling. (2020-09-11)

African wild dogs have vestigial first digit and muscular adaptations for life on the run
African wild dogs (Lycaon pictus) are known for their unique hunting style, often referred to as ''exhaustive predation'', in which they chase their prey to exhaustion, rather than hunting using speed, strength, or stealth. They are also unique among the dog clade in having only four full digits on their front paws. Until recently, it was unclear how these unique behavioral and anatomical features would affect their forelimb morphology. (2020-09-07)

Handgrip strength shown to identify people at high risk of type 2 diabetes
A simple test such as the strength of your handgrip could be used as a quick, low-cost screening tool to help healthcare professionals identify patients at risk of type 2 diabetes. (2020-09-02)

Duchenne: "Crosstalk" between muscle and spleen
Duchenne muscular dystrophy (DMD) is the most common muscle disease in children and is passed on by X-linked recessive inheritance. Characteristic is a progressive muscular atrophy. The disease often results in death before the third decade of life. Researchers of the Universities of Maynooth (Ireland) and Bonn have found a connection between dystrophic muscles and the lymphatic system in mice with Duchenne disease. The results have now been published in the journal ''iScience''. (2020-08-27)

A multicenter look at gene therapy for spinal muscular atrophy
A new study confirms the safety and efficacy of gene therapy in children with spinal muscular atrophy under two years old. (2020-08-25)

Researchers characterize important regulators of tissue inflammation, fibrosis and regeneration
Although macrophages (cells involved in the detection and destruction of bacteria and other harmful organisms as well as dead cells) are classified as immune cells functioning in the activation and resolution of tissue inflammation, it is now clear that they are critically involved in a variety of disease processes, such as chronic inflammatory diseases, tumor growth and metastasis and tissue fibrosis. (2020-08-10)

European Heart Journal: Cell infusions benefit heart patients
Three years after the ALLSTAR clinical trial ended prematurely, the study's data shows that treatments of cardiosphere-derived cells -- the same cells used as an experimental therapy tested in COVID-19 patients -- demonstrated unexpected promise in heart attack survivors. Downloadable video available. (2020-08-04)

Erectile dysfunction drugs can help cells destroy misfolded proteins
PDE5 inhibitors -- which include the erectile dysfunction drugs sildenafil and tadalafil -- can activate the cell's protein quality-control systems and improve its ability to dispose of misfolded proteins. Researchers observed lower accumulation of mutant proteins and reduced cell death and anatomical defects in zebrafish models of neurodegeneration after treatment. (2020-07-22)

NHGRI researchers generate complete human X chromosome sequence
Researchers at the National Human Genome Research Institute (NHGRI), part of the National Institutes of Health (NIH) have produced the first end-to-end DNA sequence of a human chromosome. The results, published today in the journal Nature, show that generating a precise, base-by-base sequence of a human chromosome is now possible, and will enable researchers to produce a complete sequence of the human genome. (2020-07-14)

Study: RNA repair shows promise in reversing mutations underlying a neurological disorder
Scientists successfully edited RNA in a living animal in such a way that the repaired RNA then corrected a mutation in a protein that gives rise to a debilitating neurological disorder in people known as Rett syndrome. The new study targeted and repaired the MeCP2 protein across a variety of cell types, a scientific first. (2020-07-14)

Glowing worms provide live-action movies of the body's internal scaffolding
Duke researchers have made the first time-lapse movies of the sheet-like mesh that surrounds and supports most animal tissues. While the thin layer of extracellular matrix known as the basement membrane plays key roles in development and disease, visualizing it in living organisms has been difficult to do. The team says their work offers a new way to study basement membrane defects underlying aging, and diseases ranging from cancer to diabetes. (2020-07-08)

Experimental drug shows early promise against inherited form of ALS, trial indicates
A clinical trial conducted at Washington University School of Medicine in St. Louis and elsewhere has found evidence that the experimental drug tofersen lowers levels of a disease-causing protein in people with an inherited form of amyotrophic lateral sclerosis, or ALS, caused by mutations in the gene SOD1. (2020-07-08)

New Human Gene Therapy editorial: Concern following gene therapy adverse events
Response to the recent report of the deaths of two children receiving high doses of a gene therapy vector (AAV8) in a Phase I trial for X-linked myotubular myopathy (MTM). The news ''is a tragic reminder of how difficult it is to predict outcomes in first-in-human studies (2020-07-02)

Traditional strength training vs jump training for physically inactive young adults
The aim of this study was to compare the effects of 4-weeks of Traditional Resistance Training versus Plyometric Jump Training programs on the muscular fitness of sedentary and physically inactive participants. (2020-06-30)

New treatment for common form of muscular dystrophy shows promise in cells, animals
Researchers have designed a potential new treatment for one of the most common forms of muscular dystrophy, according to a new study published today in the Proceedings of the National Academy of Sciences. Toshifumi Yokota, professor of medical genetics at the University of Alberta, led a team from Canada and the U.S. to create and test synthetic DNA-like molecules that interfere with the production of a toxic protein that destroys the muscles of people who have facioscapulohumeral muscular dystrophy (FSHD). (2020-06-29)

New treatment method for Alport Syndrome uses antisense oligonucleotides
A multi-institutional research group including Kobe University researchers has succeeded in developing an exon-skipping therapy using nucleic-acid therapeutics for Alport syndrome, an incurable kidney disease that can progress to renal failure. Disease model mice were found to be completely responsive to the treatment. It is hoped that these research achievements can contribute greatly towards the development of the world's first specialized treatment method for Alport syndrome. (2020-06-24)

New structural 'map' solves mysteries of gigantic gene regulator
Structural biology has been used to 'map' part of a protein called SMCHD1, explaining how some changes in SMCHD1 cause certain developmental and degenerative conditions. Publishing in the journal Science Signaling, the Walter and Eliza Hall Institute team revealed the structure of the portion of the SMCHD1 protein that is crucial to its function in 'switching off' genes. Inherited mutations in this part of SMCHD1 have been linked to a developmental disorder and a form of muscular dystrophy. (2020-06-17)

First report of systemic delivery of micro-dystrophin gene therapy in children with DMD
Researchers from Nationwide Children's Hospital have published in JAMA Neurology results from the first four patients treated in the first clinical trial of systemic delivery of micro-dystrophin gene therapy in children with Duchenne muscular dystrophy (DMD) -- and initial findings suggest that the therapy can provide functional improvement that is greater than that observed under the standard of care. (2020-06-15)

Treat early or wait? Experts ponder best way to manage milder forms of spinal muscular atrophy
The advent of therapeutic interventions for spinal muscular atrophy (SMA) has increased the importance of presymptomatic diagnosis and treatment. When to start treatment in children with less severe disease remains controversial. Now, in a report published in the Journal of Neuromuscular Diseases, German researchers argue for an earlier start of treatment to prevent permanent nerve damage, challenging recommendations originally proposed by a group of American experts that suggests a strict follow-up strategy for children expected to have less severe disease. (2020-06-11)

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