EDITAS MEDICINE, INC.;FONDAZIONE TELETHON;OSPEDALE SAN RAFFAELE
发明人:
COTTA-RAMUSINO, CECILLIA,MARGULIES, CARRIE M.,NALDINI, LUIGI,GENOVESE, PIETRO
申请号:
CA3079968
公开号:
CA3079968A1
申请日:
2018.10.24
申请国别(地区):
CA
年份:
2019
代理人:
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.