- 专利标题: Polynucleotides encoding engineered meganucleases having specificity for recognition sequences in the dystrophin gene
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申请号: US18360312申请日: 2023-07-27
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公开(公告)号: US12006522B2公开(公告)日: 2024-06-11
- 发明人: Gary Owens , Janel Lape , James Jefferson Smith , John Morris , Caitlin Turner , Whitney Lewis , Derek Jantz
- 申请人: Precision BioSciences, Inc.
- 申请人地址: US NC Durham
- 专利权人: Precision BioSciences, Inc.
- 当前专利权人: Precision BioSciences, Inc.
- 当前专利权人地址: US NC Durham
- 代理机构: Womble Bond Dickinson (US) LLP
- 分案原申请号: US17931896 2022.09.13
- 主分类号: C12N9/22
- IPC分类号: C12N9/22 ; C12N15/86
摘要:
The present disclosure encompasses engineered meganucleases that bind and cleave recognition sequences within a dystrophin gene. The present disclosure also encompasses methods of using such engineered meganucleases to make genetically modified cells. Further, the disclosure encompasses pharmaceutical compositions comprising engineered meganuclease proteins, or polynucleotides encoding engineered meganucleases of the disclosure, and the use of such compositions for the modification of a dystrophin gene in a subject, or for treatment of Duchenne Muscular Dystrophy.
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