Transplanting an animal organ into a human is now a closer reality following the successful xenotransplantation of a genetically modified pig liver into a patient diagnosed with brain death in China. The operation was intended to evaluate organ function over a 10-day period. This is a complex experimental trial that did not involve removing the patient's liver and still requires further study. However, the positive preclinical results suggest this strategy could save the lives of those waiting for a human organ, at least in certain cases.
In an advance that could significantly lighten the load for caretakers in the “sandwich generation” and reduce loneliness in elderly patients, Aspargo Labs Inc. developed a metered delivery device that optimizes absorption of pharmaceuticals and reminds users to take their medications.
Researchers have altered the genetic code in a strain of Escherichia coli, reducing the number of stop codons from three to one and assigning the freed-up stop codons to nonstandard amino acids. They reported on the recoded bacterium, which they named OCHRE, in Nature on Feb. 5, 2025.
Following Nobel Prize-winning chemist David Baker’s recipe for cooking an antidote to cobra venom using artificial intelligence (AI) could be faster and more effective than currently available methods. The ingredients and steps can be found in a new study published by the University of Washington scientist in collaboration with the Technical University of Denmark. They are ready for the next steps in preclinical trials.
Researchers from the University of California San Francisco (UCSF) have successfully replicated the design of regulatory T cells, achieving local targeted immune suppression and protection from CAR T-cell cytotoxicity. Many of the treatments used so far in the context of inflammatory and autoimmune disorders lead to systemic immunosuppression. In this sense, limiting immunosuppression locally to targeted tissues may help overcome systemic toxicity.
At the Breakthroughs in Muscular Dystrophy special meeting held in Chicago Nov. 19-20, 2024, and organized by the American Society of Gene & Cell Therapy (ASGCT), multiple interventions at the RNA level were among the approaches that were presented to fight muscular dystrophies.
Since the isolation of the gene that causes Duchenne muscular dystrophy (DMD), scientists have progressed in understanding the mechanisms that lead to muscular diseases that can be evident from the early stages of childhood. This has led to the development of diagnostics and therapeutics, some approved by the FDA.
An international consortium of thousands of scientists is creating the Human Cell Atlas, a three-dimensional map of all the cells in the body. The goal is to understand all the cells that make up human tissues, organs and systems, which will enable multiple medical applications. This collection of cell maps is openly available for navigation at single-cell resolution, identified through omics analyses that reveal the tridimensional distribution of each cell.
A year out from Leqembi’s approval for Alzheimer’s disease (AD), ongoing research coupled with artificial intelligence is advancing both radiopharmaceuticals and small-molecule drugs for AD diagnostics and treatment, speakers at the 2024 KoNECT-MOHW-MFDS conference said.
Gene editing strategies, from epigenetic engineering to cell reprogramming and genetic vaccines, are accelerating the development of new therapies that awaken the immune system to treat cancer, as presented last month in Rome at the 31st Annual Congress of the European Society of Gene and Cell Therapy (ESGCT). Some of these advances are taking advantage of the conditions of the tumor microenvironment, where cancer cells coexist with immune cells, microorganisms and blood vessels.