The suppression of the SYF2 factor could be a new therapeutic strategy for the treatment of the different types of amyotrophic lateral sclerosis (ALS). According to a study from the University of Southern California, SYF2 acts on the TDP-43 protein, improving the survival of motor neurons affected by this disease. “We wanted to find something that would improve neuron survival across many different iPSC lines for ALS,” Justin Ichida told BioWorld.
Researchers have developed an algorithm that was able to create functional enzymes from scratch after being trained with the amino acid sequences of existing enzymes in the same class. Researchers from the University of California at San Francisco described their method online in Nature Biotechnology on Jan. 26, 2023. The method, which its creators have named Progen, can generate “protein sequences with a predictable function across large protein families,” according to the authors.
By applying deep learning methods to a large database of zinc finger nucleases, researchers at the University of Toronto and New York University have developed an algorithm, Zfdesign, that was able to design custom zinc fingers for any given stretch of DNA. “I think this system levels the playing field for zinc fingers and CRISPR,” said Philip Kim, co-corresponding author of the team's paper published online in Nature Biotechnology on Jan. 26, 2023.
Genkore Inc. has entered into a strategic research collaboration with a U.S.-based biopharma company for the development of in vivo gene editing therapies.
An analysis of more than 1,000 small molecules has identified dozens of compounds that could be effective to treat Marfan syndrome (MFS), an inherited disorder affecting connective tissue, primarily in the heart and blood vessels, the skeleton, and the eyes. In particular, the researchers from Cambridge University found that glycogen synthase kinase-3β (GSK-3β) could be a target to develop new therapies based on its inhibition.
Bacterial abortive infection is a defense mechanism by which an infected bacterial cell enters dormancy or dies to limit phage replication and protect the clonal population. Recent studies observed that CRISPR RNA-guided adaptive immune systems that target RNA also cause abortive-infection phenotypes by activating indiscriminate nucleases.
A noninvasive method for administering monoclonal antibodies reliably and reproducibly to central nervous system (CNS) targets remains a significant unmet need. Researchers in Switzerland have reported making headway in bypassing the blood-brain barrier through the intranasal delivery of full-length antibodies in rodent models. They show this delivery route leads to rapid transfer of significant amounts of a mouse antibody targeted against Nogo-A, a membrane protein that inhibits growth of neurites in the brain and spinal cord.
Myrtelle Inc. and Raaven Therapeutics AB have partnered on the development of novel recombinant adeno-associated virus (rAAV) vectors to advance gene therapy treatments for diseases of the central nervous system (CNS) in which myelin is affected.
Kronos Bio Inc. has entered into a discovery collaboration in the field of oncology with Genentech Inc., a member of the Roche Group, focused on discovering and developing small-molecule drugs that modulate transcription factor targets selected by Genentech.