The muscular dystrophies (MD) are a group of more than 30 genetic diseases characterized by progressive weakness and degeneration of the skeletal muscles that control movement. Some forms of MD are seen in infancy or childhood, while others may not appear until middle age or later. The disorders differ in terms of the distribution and extent of muscle weakness (some forms of MD also affect cardiac muscle), age of onset, rate of progression, and pattern of inheritance.
Researchers at UF Scripps Biomedical Research have created a potential treatment for a major factor in the development of ALS and dementia that functions by removing disease-causing RNA segments.
The knowledge of what can be inherited from parents and how their life experiences impact children could be completely rewritten if a fundamental finding concerning a factor driving healthy development in embryos were made.
To celebrate National DNA Day 2022, News-Medical speaks to Jonas Korlach, Chief Scientific Officer of PacBio.
Vesicles secreted from human heart cells may repair damaged tissue and prevent lethal heart rhythm disorders, according to a new study from investigators in the Smidt Heart Institute at Cedars-Sinai.
Mutations that cause muscle atrophy can be corrected using the CRISPR-Cas9 gene-editing tool.
A clinical trial at UC Davis Health and six other sites showed that a cellular therapy offers promise for patients with late-stage Duchenne muscular dystrophy (DMD), a rare genetic disorder causing muscle loss and physical impairments in young people.
Experts at Children’s National Hospital have created a new pre-clinical gene therapy for the rare condition limb-girdle muscular dystrophy (LGMD) 2B.
An international, multidisciplinary team of researchers from the Translational Synthetic Biology Laboratory at Pompeu Fabra University (Barcelona, Spain), led by Dr. Marc Güell, has published an article in the scientific journal Nature Communications.
Researchers have created a recipe for turning skin cells into primitive muscle-like cells that can be sustained indefinitely in the laboratory.
Results of a compassionate-use study released in STEM CELLS Translational Medicine show promising results for treating muscular dystrophies with mesenchymal stem cells (MSCs) derived from Wharton's jelly (WJ), a substance found in the umbilical cord.
Researchers from UT Southwestern used a new kind of gene therapy to successfully treat mice with Duchenne muscular dystrophy (DMD), distinctively employing CRISPR-Cas9-based tools to restore a large section of the dystrophin protein missing in several DMD patients.
As the name implies, induced pluripotent stem cells can become any type of cell in our body, and scientists have evidence that when they prompt them to become muscle progenitor cells they can help restore the sometimes debilitating muscle loss that happens with age.
Researchers at Tel Aviv University have demonstrated that the CRISPR/Cas9 system is very effective in treating metastatic cancers.
Researchers have shown that the advanced CRISPR/Cas9 system is extremely effective in curing metastatic cancers.
To identify new potential therapeutic targets for SARS-CoV-2, a team of scientists at the New York Genome Center, New York University, and the Icahn School of Medicine at Mount Sinai, performed a genome-scale, loss-of-function CRISPR screen to systematically knockout all genes in the human genome.
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.
While building a muscle damage model in a cultured system, a research collaboration between Kumamoto University and Nagasaki University in Japan has found that components leaking from broken muscle fibers activate "satellite" muscle stem cells.
Gene therapy offers an excellent potential for treating specific types of genetic defects and cancer, immunological diseases, infections, and wounds.
There is great potential in gene therapy for treating certain types of cancer and genetic defects, immunological diseases, wounds and infections.
Researchers have effectively edited RNA in a living creature such that the repaired RNA subsequently rectified a mutation in a protein that leads to Rett syndrome—a debilitating neurological disorder that affects people.