(Press-News.org) Myotonic dystrophy is a hereditary degenerative neuromuscular disease that occurs mainly in adults, affecting about 50,000 people only in Spain. Symptoms range from difficulty walking and myotonia (great difficulty in relaxing the contracted muscles) to severe neurological problems, leading to progressive disability that unfortunately puts many of those affected in a wheelchair. This disease is very heterogeneous among patients (age of onset, progression, hereditary transmission, affected muscles), which makes the development of generic treatments especially complex.
Currently, drugs against myotonic dystrophy are developed in animal models such as Drosophila, the fruit fly, or in 2D cell cultures. They are later tested in mice and finally in patients. Unfortunately using these approaches, some of the drugs that have been developed have not been able to reach the last stages of clinical trials because they were not effective in patients, highlighting the difficulty of extrapolating results obtained with animals to humans.
Now, the team led by the ICREA Researcher Professor and head of the Biosensors for Bioengineering group at IBEC, Javier Ramón, and by his team's postdoctoral researcher Juan M. Fernández, has developed for the first time a three-dimensional model for myotonic dystrophy, using bioengineering techniques and patient cells. The work, which is part of the doctoral thesis of the IBEC researcher Xiomara Fernández, has recently been published in the journal Biofabrication and opens new avenues for finding personalized and effective treatments against this rare disease, and to date without cure. This work has been funded in part with funds from a fundraising campaign launched by IBEC from late 2017 to late 2019.
The new 3D model for myotonic dystrophy:
Researchers started from fibroblasts from patient's skin, which are cells of the connective tissue responsible for maintaining its structure, that were previously "reprogrammed" to become myoblasts, muscle precursor cells. These myoblasts were placed inside a gelatine and cellulose matrix and the mixture was subjected to a kind of rubber stamp to give it a shape of elongated rectangles, simulating muscle fibers. Subsequently, the matrix was incubated under the ideal conditions for the myoblasts to continue their development.
"What we observe is that, unlike classic cell cultures, the patient cells in our model are capable of fusing to form muscle fibers that adopt the 3D shape found in muscles and essential for their functioning.", states Juan M. Fernandez, researcher at IBEC.
Ability to test drugs in a personalized and fast way:
To evaluate the functionality of the new 3D model, the researchers have applied a drug, called AntagomiR-23b, to the muscle fibers. This drug has been previously developed by some authors of this work and in the not-too-distant future will enter the phase of clinical trials thanks to the Arthex Biotech company. They observed that both, the molecular and structural characteristics of the fibers derived from the patient, recovered their normal parameters when treated, validating their use for the development of new drugs.
In addition, this model has a low cost, its implementation is simple, and it can accelerate the process of developing new treatments, since it is faster compared to animal testing. This model represents a great step towards personalized medicine, since taking into account the enormous variability between patients is key when seeking treatments for this disease.
"This new model will allow testing different drugs and administering to the patients the one that has given the best results with their own cells. In this way, the treatment will be personalized, more effective and with no need for animal testing", explains Xiomara Fernandez, first author of the article.
INFORMATION:
LAWRENCE -- Like much of society, college athletics were thrown into disarray by the COVID-19 pandemic. While student athletes were suddenly prevented from competing, training or seeing as much of their teammates and coaches, those who perceived they were part of a positive sporting environment also coped better during the early days of the crisis, a new study from the University of Kansas has found.
KU researchers have long studied a caring, task-involved sporting climate, in which young athletes receive support and recognition for their efforts, while mistakes are treated as learning opportunities. But the pandemic provided a unique opportunity to see whether the approach helped collegiate athletes cope with the unique stresses and challenges that came with the disruption ...
Social media may make it easier for people to engage online, but I does not provide certain benefits of real-life human interactions, says a Michigan State University researcher.
"Problematic social media use has been associated with depression, anxiety and social isolation, and having a good social support system helps insulate people from negative mental health," said Dar Meshi, an assistant professor in the Department of Advertising and Public Relations at MSU. "We wanted to compare the differences between real-life support and support provided over social media to see if the support provided over social media could have beneficial effects."
The research was published online April 29 in the journal Addictive Behaviors.
While ...
Living materials, which are made by housing biological cells within a non-living matrix, have gained popularity in recent years as scientists recognize that often the most robust materials are those that mimic nature.
For the first time, an international team of researchers from the University of Rochester and Delft University of Technology in the Netherlands used 3D printers and a novel bioprinting technique to print algae into living, photosynthetic materials that are tough and resilient. The material has a variety of applications in the energy, medical, and fashion sectors. The research is published in ...
Nearly 10,000 acres of lush seagrass vanished from Florida Bay between 1987 and 1991, leading to massive ecological changes in the region near the Florida Keys. Abundance of the seagrass, Thalassia testudinum, more commonly known as turtlegrass, a foundation species of the Florida Bay ecosystem, decreased extensively during what is considered to be one of the largest declines in seagrass cover in recent history.
Researchers from the University of South Florida, the Florida Fish and Wildlife Conservation Commission (FWC) and the University of North Carolina Wilmington documented the response of seagrasses after the die-off. ...
31 percent of pet dogs and 40 percent of pet cats tested positive to COVID-19 after their owners' own diagnoses, though under half displayed symptoms, in small Brazilian study.
INFORMATION:
Publicly available article: https://journals.plos.org/plosone/article?id=10.1371/journal.pone.0250853
Article Title: Investigation of SARS-CoV-2 infection in dogs and cats of humans diagnosed with COVID-19 in Rio de Janeiro, Brazil
Funding: MMS: This study was supported by CGLab/MoH (General Laboratories Coordination of Brazilian Ministry of Health), CVSLR/FIOCRUZ (Coordination of Health Surveillance and Reference Laboratories ...
Thanks to the so-called deep learning, a subset of artificial intelligence (AI) algorithms inspired by the brain, machines can match human performance in perception and language recognition and even outperform humans in certain tasks. But do these synthetic biologically inspired systems learn in the same way that we do?
According to a new article by first author Dr. Diogo Santos-Pata from the Synthetic Perceptive, Emotive and Cognitive Systems lab (SPECS) at IBEC led by ICREA Professor Paul Verschure, in collaboration with Prof. Ivan Soltesz at Stanford University, the mechanism of autonomous learning underlying these AI systems reflects nature more closely than previously thought. With their hypothesis and model, these scientists offer new insights into ...
A world first study within the Great Barrier Reef Marine Park has found limited fishing zones (yellow zones) are still important conservation and fisheries management tools when paired with no-fishing zones.
Lead author Dr April Hall, from the ARC Centre of Excellence for Coral Reef Studies at James Cook University (Coral CoE at JCU), said partially protected yellow zones still contain healthy numbers of reef fish targeted for recreational and commercial fishing. These include coral trout, tropical snappers, emperors and tuskfish.
Yellow zones limit, rather than prohibit, fishing through fishing gear restrictions. For example, limited line fishing is allowed with one rod or line and one hook per ...
Bowel cancer survival rates could be improved if chemotherapy drugs were delivered via tiny nanoparticles to the diseased organs rather than oral treatment.
That's the finding from Indian and Australian scientists who have undertaken the first study, using nanoparticles to target bowel cancer, the third most common cancer in the world and the second most deadliest.
The researchers have shown in animal experiments that nanoparticles containing the chemotherapy drug Capecitabine (CAP) attach themselves directly to the diseased cells, bypassing healthy cells and therefore reducing toxic side effects as well as the size and number of tumours.
The scientists, from the Manipal Academy of Higher Education, Indian Institute of Science and the University of South Australia, ...
National Health and Medical Research Council, the European Research Council and the Victorian Government.
WEHI researchers have identified how natural human antibodies can block malaria parasites from entering red blood cells, potentially indicating how new protective therapies could be developed against this globally significant disease.
The research provides greater insight into how antibodies block the entry of Plasmodium vivax malaria parasites into young red blood cells called reticulocytes. It builds on an earlier discovery that the P. vivax latches onto the transferrin receptor 1 (TfR1) to enter cells.
The research, led by Associate Professor Wai-Hong Tham and PhD student Li-Jin Chan ...
Many drug addicts take not only one substance but rather several. Scientists at the Max Planck Institute of Experimental Medicine in Göttingen have investigated the role that genes and the environment play in the development of such multiple substance consumption or polytoxicomania. Their results show that, in addition to genetic factors, the accumulation of several unfavourable environmental factors causes people to slip into such an extreme form of multiple drug use. Among the risk factors were sexual and physical abuse, living in a big city, and migration experience as well as the use ...
