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Updated: Jun 29, 2025
A New Toolkit for Evaluating Gene Functions using Conditional Cas9 Stabilization
Published on: September 2, 2021
An Antibody-CRISPR/Cas Conjugate Platform for Target-Specific Delivery and Gene Editing in Cancer
Seungju Yang1, San Hae Im1, Ju Yeon Chung1
1Department of Biological Sciences, Korea Advanced Institute of Science and Technology, Daejeon, 34141, Republic of Korea.
Abstract:
The CRISPR/Cas system has been introduced as an innovative tool for therapy, however achieving specific delivery to the target has been a major challenge. Here, an antibody-CRISPR/Cas conjugate platform that enables specific delivery and target gene editing in HER2-positive cancer is introduced. The CRISPR/Cas system by replacing specific residues of Cas9 with an unnatural amino acid is engineered, that can be complexed with a nanocarrier and bioorthogonally functionalized with a monoclonal antibody targeting HER2. The resultant antibody-conjugated CRISPR/Cas nanocomplexes can be specifically delivered and induce gene editing in HER2-positive cancer cells in vitro. It is demonstrated that the in vivo delivery of the antibody-CRISPR/Cas nanocomplexes can effectively disrupt the plk1 gene in HER2-positive ovarian cancer, resulting in substantial suppression of tumor growth. The current study presents a useful therapeutic platform for antibody-mediated delivery of CRISPR/Cas for the treatment of various cancers and genetic diseases.
Insights
This study introduces an antibody-CRISPR/Cas conjugate for targeted gene editing in HER2-positive cancers. This innovative platform shows promise for effective cancer therapy by enabling specific delivery and disrupting tumor growth genes.
Area of Science:
- Biotechnology
- Molecular Biology
- Oncology
Background:
- CRISPR/Cas gene editing offers therapeutic potential but faces delivery challenges.
- Targeted delivery is crucial for effective cancer therapy and minimizing off-target effects.
Purpose of the Study:
- To develop an antibody-CRISPR/Cas conjugate platform for specific gene editing in HER2-positive cancers.
- To evaluate the efficacy of this platform for in vitro and in vivo cancer treatment.
Main Methods:
- Engineered CRISPR/Cas system with unnatural amino acids for nanocarrier complexation.
- Bioorthogonal functionalization with HER2-targeting monoclonal antibodies.
- In vitro and in vivo studies in HER2-positive cancer models.
Main Results:
- Successfully created antibody-conjugated CRISPR/Cas nanocomplexes.
- Demonstrated specific delivery and gene editing in HER2-positive cancer cells in vitro.
- Showed in vivo tumor growth suppression in HER2-positive ovarian cancer by disrupting the plk1 gene.
Conclusions:
- The antibody-CRISPR/Cas conjugate platform enables targeted gene editing for cancer therapy.
- This approach offers a promising strategy for treating HER2-positive cancers and other genetic diseases.
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