Supporting Data for Applying Engineered CRISPR Tools For Therapeutic Genome Editing

This dataset supports a thesis project that explores the development of two complementary CRISPR-based genome editing strategies: precision editing of a pathogenic LMNA variant and high-specificity editing of targets required for manufacturing allogeneic T cells. It features analyzed data documenting CRISPR editing efficiencies and source data from Sanger sequencing and next-generation sequencing. The cell lines studied included HEK293T and primary human T cells. These data, as described and discussed within the thesis, illustrate the potential for optimized prime editing to precisely target and correct pathogenic alleles related to congenital muscular dystrophies, and highlight how the functionally enhanced Opti-SpCas9 variant could be deployed to reduce off-target effects in a therapeutic editing application. Researchers may find these files helpful for further investigations into therapeutic CRISPR applications using base editing, prime editing, and engineered CRISPR nuclease tools.