| 发明名称 | Gene correction of SCID-related genes in hematopoietic stem and progenitor cells | ||
| 摘要 | The present disclosure is in the field of genome engineering, particularly targeted integration of a functional SCID-related genes (e.g., IL2RG, RAG1 and/or RAG2 gene) into the genome of a cell for provision of proteins lacking or deficient in SCID. | ||
| 申请公布号 | US9616090(B2) | 申请公布日期 | 2017.04.11 |
| 申请号 | US201514813924 | 申请日期 | 2015.07.30 |
| 申请人 | Sangamo BioSciences, Inc. | 发明人 | Conway Anthony;Cost Gregory J.;Holmes Michael C.;Urnov Fyodor |
| 分类号 | C12N9/16;C07H21/04;C12N5/02;A61K35/12;C12N9/22;A61K48/00;C12N15/90;A61K35/28;A61K35/545 | 主分类号 | C12N9/16 |
| 代理机构 | Pasternak Patent Law | 代理人 | Pasternak Patent Law ;Pasternak Dahna S.;Abrahamson Susan |
| 主权项 | 1. A nuclease comprising a DNA-binding domain and cleavage domain, wherein the DNA-binding domain is a zinc finger protein (ZFP), a TAL-effector domain, or a single RNA (sgRNA) and further wherein the DNA-binding domain binds to a target site in intron 1 of an endogenous IL2RG gene or intron 1 or 2 of an endogenous RAG gene such that the nuclease cleaves the IL2RG or RAG gene. | ||
| 地址 | Richmond CA US | ||