2023 56 B3

Preventing Germline Genome Editing with miRNA-tagged Payload

By: Travis McCreary

Department: Biology

Faculty Advisor: Dr. Lily Chen

In-vivo editing has one major outcome. That the on-target editing occurs in the desired cell type. Due to a recent death of a patient in a CRISPR clinical trial, demands us to question the safety of non-targeted delivery strategies which may lead to life-threatening immune reactions. Given these concerns, there is a major unmet clinical need to develop targeted in-vivo delivery that is specific to desired cell types. This is what creates the need for editing tools that can prevent activity in undesired cell types. The hypothesis is that the delivery of Germline specific miRNA-tagged payload will result in reduced gene expression in germline cells. Delivering the same miRNA-tagged payload to HSCs will result in higher expression than germline cells.