A landmark study published in Cell has shown that prime editing, a cutting-edge form of gene editing, can correct mutations causing Alternating Hemiplegia of Childhood (AHC) with a single in-brain injection. The research team fixed the most prevalent ATP1A3 gene mutations in mouse models, reducing symptoms and more than doubling survival, a first-of-its-kind success in treating a neurological disease directly in the brain. CRISPR-based gene editing was delivered through an harmless adeno-associated virus called AAV9. In parallel, patient-derived cells (iPSCs) responded similarly, reinforcing the method’s promise for human translation. Importantly, this success opens the door to targeting other genetic brain disorders previously deemed untreatable. Although results are preliminary, this study provides robust proof‑of‑concept for personalized gene editing in the brain and opens doors toward potential treatments for other intractable genetic neurological disorders.
In a recent study published in the journal Nature, researchers have compiled the available literature on natural killer (NK) cells, innate immune cells involved in the recognition and elimination of cells in distress, particularly virus-infected cells and tumors. They focus on reviewing current preclinical and clinical research in the field of NK therapies, primarily elucidating the role of NK cells in cancer immunity. They also explore the potential of bioengineering approaches to harness NK cells via the development of genetically modified NK cells, immune checkpoint inhibitors and cell engagement agents. The study reveals that, despite less than two decades of research in the field, NK cells are emerging as a safe, practical and potentially widely accessible means of clinical therapy, particularly antitumor. Although challenges exist in the adoption of NK cell therapies by conventional medicine, studies aimed at overcoming these challenges are already underway, bringing the future of clinical NK cell interventions closer than ever.