专利名称 | SYSTEMS AND METHODS FOR TREATING HYPER-IGM SYNDROME | ||
申请号 | WOUS18057354 | 申请日 | |
公开(公告)号 | WO2019084168A1 | 公开(公告)日 | |
申请(专利权)人 | EDITAS MEDICINE INC; FONDAZIONE TELETHON; OSPEDALE SAN RAFFAELE | 发明人 | COTTA RAMUSINO Cecillia; MARGULIES Carrie M; NALDINI Luigi; GENOVESE Pietro |
专利来源 | 国家知识产权局 | 转化方式 | |
摘要 |
Disclosed herein are genome editing systems and related methods which allow for treatment of Hyper IgM Syndrome, a group of disorders characterized by defective CD40 signaling. The compositions and methods described herein rely on the use of donor templates comprising a CD40L exons to restore proper CD40 signaling and B cell class switch recombination. |
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