基本信息:
- 专利标题： THERAPEUTIC GENOME EDITING IN X-LINKED HYPER IGM SYNDROME
- 申请号：US17050601 申请日：2019-04-24
- 公开(公告)号：US20210324381A1 公开(公告)日：2021-10-21
- 发明人： David J. Rawlings , Daniel Thomson , Iram F. Khan
- 申请人： Seattle Children's Hospital (dba Seattle Children's Research Institute)
- 申请人地址： US WA Seattle
- 专利权人： Seattle Children's Hospital (dba Seattle Children's Research Institute)
- 当前专利权人： Seattle Children's Hospital (dba Seattle Children's Research Institute)
- 当前专利权人地址： US WA Seattle
- 国际申请： PCT/US19/28858 WO 20190424
- 主分类号： C12N15/11
- IPC分类号： C12N15/11 ; C12N9/22 ; C12N15/90 ; C07K14/705 ; C12N15/87 ; C12N15/86 ; A61K38/46 ; A61K31/7088
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.