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.

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.

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.

Esperovax Inc. and Ginkgo Bioworks Inc. have established a partnership to develop circular RNAs (circRNAs) for a variety of therapeutic applications.

CRISPR, or clustered regularly interspaced palindromic repeats, is transforming biomedical research, and making rapid inroads into the clinic, with its ability to easily target specific DNA and RNA sequences. CRISPR itself is made of RNA. It recognizes target sequences and delivers CRISPR-associated (Cas) proteins, nucleases that cut the target sequence. In two papers published online in Nature on Jan. 4, 2023, researchers have demonstrated that a recently discovered type of Cas protein, Cas12a2, can degrade double-stranded DNA when its associated CRISPR guide RNA recognizes its target sequence.