Although there are different methods of nuclear gene editing, there are still no effective treatments against mitochondrial disorders due to genetic alterations. Now, a group of researchers at Precision Biosciences Inc. and the University of Miami (UM) has developed a genetic edition platform that targets mitochondrial DNA (mtDNA) to delete its mutations.
“The ARCUS technology that we use is based on an enzyme found in nature called I-CreI. It is an enzyme that recognizes a 22 base pair DNA sequence within a species of green algae. And when it finds that DNA sequence, it will generate double-strand breaks,” first author Wendy Shoop, a scientist at Precision Biosciences, told BioWorld.
Terray Therapeutics Inc. has established a multi-target collaboration agreement with Bristol Myers Squibb to discover and develop small-molecule therapeutics in certain disease areas.
Researchers have developed a new approach for the development of improved CAR T cells with bifunctional degraders, which linked ubiquitin to an endogenous target protein. The key to the design was the use of multispecific protein degraders and E3 ligases, which increased the proliferation of CAR T cells and their antitumor potency. This combination can be adapted to different uses of cell therapies.
Researchers from New York University College of Dentistry’s Pain Research Center in collaboration with the University of Pittsburgh found a candidate for chronic pain treatment among a library of 27 million compounds.
Novel approaches are needed to improve anti-inflammatory drug delivery to the brain, particularly to glial cells, in order to reduce neuroinflammation.
Postera Inc. has announced a multitarget collaboration with Amgen Inc. that seeks to leverage Postera’s artificial intelligence (AI) platform, Proton, an innovation in generative chemistry and synthesis-aware design, and Amgen’s drug discovery expertise, to advance up to five small-molecule programs.
A new derivative of coumarin, a natural plant product abundant in cinnamon, could hold the key to healthy aging. Researchers at the Buck Institute have shown that it extended life span and prevented neurodegenerative disease in worms and mice. The drug, a TFEB gene inducer called MIC, promoted mitochondria recycling (mitophagy) but also interacted with lysosomes, which could have multiple applications. The scientists published the results of this aging and mitophagy study on Nov. 13, 2023, in Nature Aging.
Investigators at the Chinese Academy of Sciences have generated a chimeric monkey by injecting an embryonic stem cell into the morula, which is an extremely early embryo consisting of 16 to 32 cells. The animal survived for only 10 days, and it is not the first live birth of a chimeric primate. But it is the first such chimera with contributions from an embryonic stem cell, and that stem cell contributed a far higher proportion of cells in the newborn than have been achieved in previous attempts at creating chimeras.