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THERAPEUTIC GENOME EDITING IN X-LINKED HYPER IGM SYNDROME
THERAPEUTIC GENOME EDITING IN X-LINKED HYPER IGM SYNDROME
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机译:X连锁超级IGM综合征的治疗性基因组编辑
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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.
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