Engineered TSC2
    2.
    发明授权

    公开(公告)号:US11639508B2

    公开(公告)日:2023-05-02

    申请号:US16631069

    申请日:2018-07-13

    IPC分类号: C12N15/85 A61K35/17

    摘要: Provided herein are engineered TSC2 polypeptides, and nucleic acid sequences encoding them, in which the ability of a serine residue to be phosphorylated is altered. In some aspects, the TSC2 serine residue cannot be phosphorylated (e.g., by substituting the serine residue with an alanine residue). In some aspects, the TSC2 serine acts as if it is constitutively phosphorylated (e.g., by substituting the serine residue with a glutamic acid residue). Also provided herein are engineered immune cells comprising altered TSC2 polypeptides or nucleic acid sequences encoding them, and methods of making and using such engineered immune cells.

    OPTIMIZED CRISPR/CAS9 SYSTEMS AND METHODS FOR GENE EDITING IN STEM CELLS

    公开(公告)号:US20230126434A1

    公开(公告)日:2023-04-27

    申请号:US17836165

    申请日:2022-06-09

    摘要: The methods and compositions described herein surprisingly increase CRISPR/Cas-mediated gene editing in stem cells by transiently treating the cells with a stem cell viability enhancer prior to and/or after contacting the cells with one or more CRISPR/Cas9 components. Further, this treatment also surprisingly results in increased engraftment of the stem cells into the target tissue of a subject. The present disclosure also provides one or more modified CRISPR/Cas9 components which, when used in combination with the stem cell viability enhancer, further increases the frequency of gene editing in stem cells, increases stem cell viability, and increases stem cell engraftment.