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公开(公告)号:US20230279440A1
公开(公告)日:2023-09-07
申请号:US17996701
申请日:2021-05-06
申请人: CELLECTIS S.A.
发明人: Alexandre JUILLERAT , Philippe DUCHATEAU , Patrick HONG , Laurent POIROT , Brian BUSSER , Alex BOYNE
CPC分类号: C12N15/907 , C12N15/86 , C12N2750/14143 , C12N2830/42
摘要: The present disclosure provides methods to genetically modify cells by insertion of an artificial exon (ArtEx) for delivery of therapeutic proteins in specific cell types and more particularly engineered cells for expression of a transgene into the brain of a patient.