Researchers who follow their instincts and achieve slow results while trying to break barriers have little support. They replace it with persistence. This is the story of Katalin Karikó and Drew Weissman. What was once a dream in their minds was later a success. Their work together for decades was essential to achieving mRNA vaccines, and their perseverance was rewarded today with the 2023 Nobel Prize in Medicine.

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.

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.

Scientist Ian Wilmut, who led a team from Scotland’s Roslin Institute and biotech company PPL Therapeutics plc to clone Dolly the Sheep in 1996, died on Sept. 10 at age 79. Dolly was the first mammal cloned from an adult cell taken from the mammary gland of a 6-year-old Finn Dorset sheep and an egg cell from a Scottish Blackface sheep.

“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.