Applying Genome Editing to Treat Congenital Insensitivity to Pain

Protective pain perception is an essential physiological mechanism that alerts the organism to tissue damage and injury. Congenital insensitivity to pain (CIP) is a rare condition, with multiple genetic causes, in which patients have severely impaired pain perception. Although CIP does not inherently shorten lifespan, it can lead to a number of secondary health risks that may impact life expectancy and quality of life. There is currently no cure for CIP, and treatment is therefore limited to patient education. We will develop efficient in vivo base- and prime-editing strategies in the nervous system to treat a CIP mouse model that mirrors human symptoms. This work could pave the way toward future therapies for CIP patients.