The incidence of central nervous system (CNS) disease has persistently increased over the last several years. There is an urgent need for effective methods to improve the cure rates of CNS disease. However, the precise molecular basis underlying the development and progression of major CNS diseases remains elusive. A complete molecular map will contribute to research on CNS disease treatment strategies. Emerging technologies such as single-cell RNA sequencing (scRNA-seq) and Spatial Transcriptomics (ST) are potent tools for exploring the molecular complexity, cell heterogeneity, and functional specificity of CNS disease. scRNA-seq and ST can provide insights into the disease at cellular and spatial transcription levels. This review presents a survey of scRNA-seq and ST studies on CNS diseases, such as chronic neurodegenerative diseases, acute CNS injuries, and others. These studies offer novel perspectives in treating and diagnosing CNS diseases by discovering new cell types or subtypes associated with the disease, proposing new pathophysiological mechanisms, uncovering novel therapeutic targets, and identifying putative biomarkers. Graphical Abstract Implications of scRNA-seq and ST for CNS diseases. A comprehensive understanding of cellular information and spatial context using scRNA-seq and ST, helping to elucidate the biological functions, the intricate connection between crucial regulatory pathways, and cell communication after CNS diseases.

Ocrelizumab, a humanized anti-CD20 monoclonal antibody approved for multiple sclerosis in 2017, modulates B-cell activity to reduce disease progression. While clinical trials demonstrate its efficacy, real-world safety data remain critical to identify rare or long-term adverse events (AEs) not captured in controlled studies. This study analyzed post-marketing AEs of ocrelizumab using data from the U.S. Food and Drug Administration Adverse Event Reporting System (FAERS) to assess safety under real-world conditions. A retrospective pharmacovigilance analysis was conducted on FAERS data from Q1 2017 to Q4 2023. Disproportionality analyses including reporting odds ratio, proportional reporting ratio, Bayesian Confidence Propagation Neural Network, and Multi-item Gamma Poisson Shrinker were used to detect safety signals. Subgroup, sensitivity, and time-to-onset analyses further characterized AE profiles. Among 50,967 reports, the analysis confirmed several AEs consistent with the ocrelizumab prescribing information, including urinary tract infection, depression, cough, herpes zoster, and breast cancer. Furthermore, potential new safety signals not previously documented in the drug’s label were identified, such as alopecia, insomnia, weight increased, and sepsis. This study confirms known AEs and identifies unexpected AEs, emphasizing the need for targeted monitoring, particularly during early therapy. Real-world FAERS data complement trial findings, aiding clinicians in optimizing multiple sclerosis treatment strategies.