Researchers delivered the ABE8e-V106W base editor as a protein into fertilized human embryos and successfully edited all copies of the PCSK9 gene without producing insertions or deletions, allowing embryos to develop to the blastocyst stage and yielding homozygous edited stem cell lines. However, they also observed occasional chromosome breaks, mosaic editing at nearby and off-target sites, and frequent embryo arrest when the editor was delivered as mRNA instead of protein.
Scientists have developed a drug delivery approach that targets specific structures within individual cells rather than simply reaching a cell as a whole. This subcellular precision aims to route therapeutic payloads to particular organelles or compartments where they can act most effectively.
Scientists are developing intranasal vaccines designed to trigger immunity directly in the nose and upper airway, the entry point for many respiratory pathogens. Unlike injected vaccines that mainly prevent severe illness, these formulations aim to stop infection and transmission before a virus can spread further.
Researchers are redesigning drug molecules and delivery systems so a single injection or implant can maintain therapeutic effects for weeks, months, or even years instead of requiring daily dosing. Techniques include chemical modifications that slow drug breakdown, polymer depots, and implantable devices that release medication gradually over extended periods.
A Nature piece argues that even well-designed drugs fail patients if they cannot reach the intended biological target effectively. The commentary highlights delivery mechanisms and targeting accuracy as critical, often underappreciated, factors in therapeutic success.
A Nature editorial argues that a substantial share of prescribed medicines fail to help patients simply because they are not taken as directed. It calls for more research and health-system attention on why patients skip or stop treatments, rather than focusing solely on drug development.
Scientists are engineering particle-based delivery systems intended to ferry large molecules such as proteins and messenger RNA past the blood-brain barrier, a membrane that normally blocks such compounds from reaching brain tissue. Small chemicals like paracetamol already pass through naturally, but bigger therapeutic molecules cannot, limiting treatment options for brain disorders.
Researchers at the University of Minnesota, led by surgeon Daniel Saltzman, have developed a therapy using genetically modified Salmonella Typhimurium bacteria to carry interleukin-2, an immune-stimulating molecule, straight into tumours. Pancreatic cancer patient John Heller received the experimental treatment after starting chemotherapy for advanced disease, illustrating an early real-world test of the approach.
A Nature feature surveys six novel approaches under development for delivering drugs more precisely and effectively, ranging from advanced nanoparticle carriers to devices designed to target specific tissues. These innovations aim to overcome longstanding challenges such as poor bioavailability, degradation before reaching target sites, and off-target side effects.
Scientists are developing genetically modified bacteria designed to seek out and treat tumors from within the body. The approach relies on engineering microbes to recognize cancerous tissue and deliver therapeutic effects directly at the tumor site.
A new speculative fiction piece from Nature Futures follows Melba, an employee managing the Lost Luggage Department at a nearly abandoned interstellar flight headquarters. The story details how travelers' bags are shrunk via molecular-reduction technology before space voyages, and how some end up misrouted for decades, eventually landing in Melba's basement office filled with stacks of miniaturized suitcases.
Google DeepMind's Demis Hassabis proposed training a large language model only on scientific knowledge available before 1911 to see if it could independently derive general relativity, calling it a meaningful test for artificial general intelligence. Several research teams, including one led by Ido Kaminer at Technion, have since built such 'vintage' AI models restricted to historical data, but early results have exposed shortcomings in current systems rather than demonstrating breakthrough reasoning.
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