Investigators at Cold Spring Harbor Laboratory have discovered a method of switching cancerous rhabdomyosarcoma cells back into normal muscle cells that could lead to the development of a treatment for this rare aggressive childhood cancer.
Research led by investigators at Ghent University in Belgium showed dysregulation of the complement part of the immune system, regulated by the pro-inflammatory protein interleukin (IL)-6, is a key driver of severe COVID-19 and a good target for drugs to treat the effects of the disease. Writing in the Aug. 23, 2023, issue of Science Translational Medicine, the researchers also described a cellular map of the alterations seen in the complement system during COVID-19 related respiratory deterioration for use in future research.
Research in rhesus monkeys has shown a gene therapy that enhanced activity in dopamine producing neurons in the brain was effective at stopping excessive alcohol consumption in previously addicted animals.
The composition of the skull bone is unique and plays a direct role in influencing brain health through small channels in the bone and immune cell expression pathways, reported researchers based at the Helmholtz Center and Ludwig-Maximilians University Munich.
The composition of the skull bone is unique and plays a direct role in influencing brain health through small channels in the bone and immune cell expression pathways, reported researchers based at the Helmholtz Center and Ludwig-Maximilians University Munich.
Researchers at the Salk Institute for Biological Studies have shown a possible beneficial role for muscle wasting in mice infected with Trypanosoma brucei, a parasite that causes sleeping sickness in humans. The team assessed the impact of both fat and muscle wasting on survival of mice with T. brucei infection. They found that while fat wasting appeared to have no impact on survival, mice that were unable to undergo muscle wasting died more quickly from the infection than those that experienced muscle loss.
Researchers based at Northwestern University Feinberg School of Medicine have discovered that a lack of contact between mitochondria and lysosomes in the cells of patients with a genetic form of Parkinson’s disease likely contributed to their symptoms.
Research led by Columbia University investigators has shed light on the immune pathway of the intestinal damage caused in celiac disease after gluten consumption. As reported in the July 14, 2023, issue of Science Immunology, the investigators showed an important role for cytotoxic T cells in addition to gluten-specific CD4+ T cells in the onset of celiac disease symptoms in a range of people with the condition.
Research led by St. Jude Children’s Research Hospital and Harvard University shows base-editing approaches could be more effective than CRISPR-Cas9 gene-editing approaches for treating conditions such as sickle cell disease and β-thalassemia. Writing in the July 3, 2023, issue of Nature Genetics, the researchers compared three base-editing approaches with two CRISPR-Cas9 approaches to increasing levels of fetal hemoglobin in CD34+ hematopoietic stem and progenitor cells, and found one of the base-editing approaches was the most potent.
New research shows base and prime editing can correct some forms of phenylketonuria (PKU) in mice and human cell lines, raising the prospect that this gene-editing approach could allow children born with the inherited metabolic disorder to have a treatment that would avoid the need for dietary restrictions and medication.