Minimal Dataset for Characterization of a C9orf72 Knockout Danio rerio Model for ALS and Cross-Species Validation of Therapeutics in Caenorhabditis elegans.

This dataset contains raw and processed data supporting the generation and characterization of a C9orf72 loss-of-function (LOF) zebrafish (Danio rerio) model and a complementary Caenorhabditis elegans (alfa-1) LOF compound screening platform. The dataset comprises numerical, image-derived, and validation data from molecular, behavioural, survival, and morphological assays performed across defined genotypes, treatments, and developmental stages.For zebrafish, the dataset includes CRISPR/Cas9 target validation data (sequencing chromatograms and off-target analyses), quantitative gene expression measurements, western blot antibody validation, larval survival and growth assays, automated locomotor activity tracking under multiple dark–light paradigms, spinal motor neuron morphology and branching analyses, neuromuscular junction integrity quantification, and pharmacological treatment experiments. For C. elegans, the dataset includes swimming activity measurements, paralysis assays, and neurodegeneration assessments following treatment with multiple compounds, including melatonin and pizotifen malate.Data files are organized by organism, experimental assay, genotype, treatment condition, and developmental stage. Both raw data and processed summary tables used for statistical analyses and figure generation are provided in open, reusable formats, with detailed metadata describing variables, units, normalization procedures, sample sizes, and experimental group definitions. Representative raw files and analysis objects are included where relevant to support data provenance and reproducibility.This dataset is associated with the manuscript “Characterization of a C9orf72 Knockout Danio rerio Model for ALS and Cross-Species Validation of Potential Therapeutics Screened in Caenorhabditis elegans”, currently under review at PLOS ONE, and provides the numerical data required to reproduce the analyses reported therein. This dataset contains no human subjects information, and all experiments were conducted in accordance with institutional animal care and ethical guidelines. The dataset may be reused for comparative analyses of C9orf72 loss-of-function phenotypes, cross-species therapeutic validation, and methodological development in neurodegeneration research.