Optimization of Automated High-Throughput CRISPR Screening Platform and Its Application in Rare Metabolic Diseases
Abstract
screening platforms are reviewed. Briefly, recent progress in pooled screening, single cross-cell screening, high content screening, in vivo
screening, and organoid screening are summarized. Coupled with the molecular properties of rare metabolic diseases, application value of this
platform in pathogenic gene identification, modifying factor mining, metabolic pathway analysis, drug target discovery, and patient model research is discussed. Other factors such as off-target effects, difficulty to quantify complex phenotypes, lack of realism in disease models, multidimensional data integration, and multidose imaging are also discussed. Furthermore, high specificity editing tools, combination of single-p
and spatial omics, organoid, and clinical translation are discussed.
Keywords
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DOI: http://dx.doi.org/10.70711/frim.v4i6.9670
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