INTRACELLULAR ANTIBODY AND PREPARATION METHOD AND USE THEREOF

    公开(公告)号:US20240343785A1

    公开(公告)日:2024-10-17

    申请号:US18611706

    申请日:2024-03-21

    Abstract: Disclosed are an intracellular antibody (intrabody) and a preparation method and a use thereof. The intrabody includes a fragment of the variable region of the heavy chain (VH) of a monoclonal antibody that recognizes mutant huntingtin (mHTT) and a signal sequence of lysosome-associated membrane protein I (LAMP1). The VH fragment includes an amino acid sequence as shown in any one of SEQ. ID NO. 1-3. The intrabody described in this application possesses a bidirectional recognition function, enabling it to bind to and recognize mHTTs as well as specifically recognize lysosomes, thereby realizing the targeted degradation of mutant proteins and improving the degradation efficiency of the mutant proteins.

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