Hot topics in Cellular Neuropathology IV—targeting neurological diseases by gene therapy approaches

enzymatic machinery, Charpentier and Doudna reprogrammed these molecular scissors, enabling them to cleave DNA molecules at predetermined sites (Jinek et al., 2012). Extended to eukaryotic cells, this technique has revealed unprecedented possibilities in gene editing, profoundly augmenting our comprehension of cellular physiological and pathological processes. For their discovery, both scientists were awarded with the Nobel Prize in Chemistry in 2020.Gene editing and therapy have emerged as a cornerstone of modern biotechnology over the past decade. Its global utilization across bioscience domains underscores its impact and significance, and it applications have extended far into the field of clinical neurosciences. So far, gene editing and therapy has inseminated Cellular Neuropathology in the following ways:• Treating inherited neurodegenerative diseases: Gene therapy technology holds promise for treating inherited neurodegenerative diseases in the nervous system. Researchers have been exploring its potential for correcting mutations associated with neurodegenerative diseases such as spinal muscular atrophy (SMA) (Mendell et al., 2017;Day et al., 2021) and Huntington's disease (Morelli et al., 2023;Dolgin, 2025), bringing gene therapy strategies into clinical practice. In randomized controlled studies, gene therapy approaches revealed striking therapeutic efficacy in disease areas, which so far seemed untreatable (Mendell et al., 2017;Day et al., 2021).• Modeling Neurological Disorders: Gene editing, including CRISPR/Cas9, has been used to generate cellular and animal models of various neurological disorders. This allows scientists to study disease mechanisms more accurately, potentially leading to insights into the underlying causes of disorders like Alzheimer's disease (Schrauben et al., 2020), Parkinson's disease (Yang et al., 2019), and autism (Elamin et al., 2023).• Understanding Neurodevelopment: By precisely manipulating genes in developing organisms, researchers have been able to uncover the roles of specific genes in brain development (Zhang et al., 2018). This knowledge can shed light on conditions like intellectual disabilities and developmental disorders. Three-dimensional induced pluripotent stem cell (iPSC) organoids have recently attracted great interest in the analysis of human brain development and pathology (Jain et al., 2025). Human brain organoids offer unprecedented possibilities for evaluating gene therapy strategies.• Functional Genomics: Gene technology has enabled scientists to perform large-scale functional genomics studies in neural cells (Sandberg et al., 2018), identifying genes that play key roles in neural function and connectivity. Dirk M. Hermann: Hot topics in Cellular Neuropathology IV / 5 This could provide valuable insights into conditions involving altered neural circuits.• Regenerative Medicine: Gene editing technology has been investigated for promoting neuronal regeneration after CNS injuries (Keatinge et al., 2021).Targeted gene editing could potentially stimulate the growth and repair of damaged neural tissue.In an effort to identify the most promising concepts in translational neurosciences, the Cellular Neuropathology section of Frontiers in Cellular Neurosciences recently launched a platform, the Hot Topics hub (Hermann, 2020). Within this platform, this journal searches for impactful papers in Cellular Neuropathology, which carry landscape-changing potential, broaden imagination horizons and expand current diagnostic or therapeutic possibilities. Within this platform, previous Research Topics evaluated the role of subtle neuroinflammation in chronic neurodegeneration (Hermann et al., 2022), the role of neuronal plasticity in the injured CNS (Hermann et al., 2023) and the role of gene editing in understanding neurological diseases (Hermann, 2023).