This work is focused on the development of potential therapeutic approaches to treat genetic disorders. The researchers employ gene- and base editing to correct disease-causing mutations and knock out or modify the expression of genes to prevent disease. In addition, they use viral vectors to deliver therapeutic genes. The target cell populations for most of the group's ex vivo cellular therapies are terminally differentiated and stem cell populations of the hematopoietic system. They use next-generation sequencing to verify the introduced genetic changes and to detect undesired changes at other locations in the genome. They are also starting to use RNA-seq and scRNA-seq to determine differential expression of diseased versus wild type cells and tissues.