Researchers have ameliorated both monogenic and complex inflammation-driven diseases through transplantation of hematopoietic stem cells with an inserted IL-1 receptor antagonist (IL-1RA) gene. The team showed that in animal models the transplanted cells worked better than monoclonal antibodies to reduce symptoms in systemic autoinflammatory diseases (SAIDs), a group of childhood-onset, lifelong diseases that vary in severity depending on the underlying mutation, but can be life-threatening.
Gene therapy technology makes it possible to select diseased or mutated cells from a patient, modify them in the laboratory and reintroduce them to the body to treat different disorders. This is known as ex vivo autologous gene therapy. The difference with allogeneic cell techniques is whether the donor is oneself (autologous) or a compatible person (allogeneic), which would provide healthy cells that do not need genetic modification.
Cells of Saccharomyces cerevisiae, a yeast used as a model for human mitosis, age in two ways. Both genomic instability and the decline of mitochondria cause cells to degenerate and die. The choice of one type or another depends on a network of genes that can be adjusted by bioengineering.
Grünenthal Gmbh and King’s College London have entered into a 24-month collaboration to develop microfluidic culture (MFC) models based on human induced pluripotent stem cells (iPSCs) for pain research.
Base editing (BE), a technique that modifies a single nucleotide in living cells, has been successfully tested to resolve the CD3δ mutation in severe combined immunodeficiencies (SCIDs) and produce functional T cells. For now, scientists at the University of California, Los Angeles (UCLA), completed the study on patient stem cells and artificial thymic organoids, shortening the way for future clinical trials.
Researchers have linked Duchenne muscular dystrophy (DMD) to a loss of regenerative capacity of muscle stem cells. The findings, which were published in the March 1, 2023, issue of Science Translational Medicine, suggest that boosting the regenerative capacity of muscle stem cells could delay or perhaps even prevent the progression of DMD. DMD is “an early and horrible disease,” senior author Frederic Relaix, who is the director of a research team studying the biology of the neuromuscular system at the Mondor Institute for Biomedical Research told BioWorld.
An international team of researchers has created two bat stem cell lines that reveal an unusual number of viral sequences in bat cells compared with those of other mammals. Writing in an article posted online Feb. 21, 2023, in Cell, the scientists suggested that the unusual amount of viral genetic material found in the bat stem cells could explain why these mammals are largely unaffected by most viral infections, despite being able to transmit them.
Using single-cell RNA sequencing of deer antler at different stages of their annual cycle of regeneration, Chinese researchers have identified a progenitor cell population that drove antler regeneration. The authors of an accompanying editorial wrote the findings, which were published in the Feb. 24, 2023, issue of Science, “add to the emerging idea that blastema progenitor cells are a common stem cell type in mammalian appendage regeneration.”