THERAPEUTIC GENOME EDITING IN X-LINKED HYPER IGM SYNDROME

摘要

Described herein are compositions, systems, and methods for treating, inhibiting, or ameliorating X-linked hyper IgM syndrome (X-HIGM) in subjects that have been identified or selected as being ones that would benefit from a therapy to treat, inhibit, or ameliorate X-HIGM. The systems include nuclease and vector donor constructs configured for co-delivery to modify endogenous CD40LG locus.