A new gene editing method uses the CRISPR technique to modify the cells of an organ in vivo, creating a mosaic used to identify the effects of each altered gene. Scientists from the Swiss Federal Institute of Technology (ETH) in Zürich developed this technology called AAV-Perturb-seq, based on adeno-associated virus (AAV) to target, edit and analyze single-cell genetic perturbations.

Arialys Therapeutics Inc. launched this month with $58 million in seed funding, an experimental compound it is developing for autoimmune encephalitis and autoimmune psychosis, and high aspirations for its field. “Yes, I want to treat these patients, I want these patients to have a better life. But I also want drug discovery and development folks to think differently about discovering new drugs for the CNS,” Jay Lichter told BioWorld.

Proteome analysis with artificial intelligence has made it possible to create a catalog of all possible missense mutations in the human genome to predict diseases. The new Alphamissense tool from the technology company Google Deepmind, available online, will allow scientists to refine diagnoses and design more tailored treatment strategies for patients suffering from pathologies associated with these variants.

By creating a new mouse model of Alzheimer’s disease that better recapitulated how the disease plays out in humans, investigators at KU Leuven have gained new insights into how amyloid plaques, tau tangles and neuronal death are related at the molecular level.

“I am not a fortune teller, nor am I a gambler. I will make no bets,” Lorraine Kalia told the audience at the 2023 International Congress of Parkinson’s Disease and Movement Disorders. “But I am optimistic.” At the meeting, which is being held in Copenhagen this week, Kalia, who is a scientist at Toronto Western Hospital’s Krembil Brain Institute and at the University of Toronto’s Tanz Centre for Research in Neurodegenerative Diseases, was giving an overview of “Emerging targets in the clinic” in a plenary session on “Therapeutic strategies for the future.”

KRAS-mutated tumors were once untreatable. In fact, KRAS was something of a poster child for so-called undruggability. Several laboratories are investigating strategies to address other mutations and uses beyond non-small cell lung cancer (NSCLC) and colorectal cancer. If you can't bind KRAS to block it, use a glue or combine multiple weapons. This is the idea behind two new approaches that target cancers caused by this proto-oncogene.

A newly discovered antibiotic has been shown to block the synthesis of bacterial cell walls via immutable targets, raising the prospect of a class of drugs that will not lose effect through the development of antimicrobial resistance. Clovibactin, isolated from soil bacteria, targets the cell wall precursor molecules lipid II, lipid III and undecaprenyl phosphate (C55PP), all of which have a pyrophosphate group in common.

The vast variety of tumors makes each cancer a world. For researchers, understanding the commonalities and divergences in their molecular underpinnings could help find successful treatments. Scientists from the Clinical Proteomic Tumor Analysis Consortium (CPTAC) have addressed these similarities and differences in 10 different types of cancer with two proteogenomic studies to unravel the genes that lead to cancer and the galaxy of interactions that regulate them.

Scientists have discovered that a small chemokine protein released by activated platelets, platelet factor 4 (PF 4), reduced neuroinflammation, and improved cognition in aged mice. The study was published on Aug. 16 in the online edition of Nature.