About the lab

Conklin’s lab is focusing on using CRISPR-related technology to treat incurable genetic diseases. By focusing on human mutations that are highly penetrant, we use patient-specific iPSCs to derive the disease tissue, and model genome surgery, prior to human clinical trials. We use whole-genome sequencing to identify common genetic polymorphisms, which can be used to selectively inactivate the disease allele with CRISPR nucleases. The diseased cell types allow us to decode the cellular signatures of disease and determine if the excision of the disease allele restores cellular functioning.