Resource Core III: Biological Impacts Core

PROJECT SUMMARY / ABSTRACT The mission of “Resource Core III: Biological Impacts Core” is to provide state-of-the-art biochemical and molecular pathology tools to the research projects supported by the Correction of Neurological Disease via Allele Specific Excision of Pathogenic Repeats (CASEPR) program. Complementing the work of Cores I and II, the Bio Impacts Core III will play an integral role in assessing the molecular phenotype of animal models following in vivo genome editing. The Core will develop innovative new assays to evaluate the impact of targeted CRISPR- mediated excision of pathogenic alleles on the transcriptome and proteome of neurons, glia, and resident- immune cells in the mammalian brain and circulatory system. Research support services include nucleic acid and protein isolation, quantitative PCR and western blot, automated microscopy, post-image analysis and quantification, immunological assays, and data management. All samples will be tracked and stored using a laboratory management system, and results disseminated back to the research project leads. Research output from Core III will provide essential data on both the efficacy and safety of the genome editing approaches; and the suite of services will accelerate the timeline from preclinical studies to IND by consolidation of resources and expertise.