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Molecular and Immunologic Techniques in a Genetically Engineered Mouse Model of Gastrointestinal Stromal Tumor
Published on: May 2, 2022
Anti-KIT DNA Aptamer for Targeted Labeling of Gastrointestinal Stromal Tumor
Sudeep Banerjee1,2, Hyunho Yoon1, Mayra Yebra1
1Division of Surgical Oncology, Department of Surgery, Moores Cancer Center, University of California, San Diego, La Jolla, California.
Abstract:
Gastrointestinal stromal tumor (GIST), the most common sarcoma, is characterized by KIT protein overexpression, and tumors are frequently driven by oncogenic KIT mutations. Targeted inhibition of KIT revolutionized GIST therapy and ushered in the era of precision medicine for the treatment of solid malignancies. Here, we present the first use of a KIT-specific DNA aptamer for targeted labeling of GIST. We found that an anti-KIT DNA aptamer bound cells in a KIT-dependent manner and was highly specific for GIST cell labeling in vitro Functionally, the KIT aptamer bound extracellular KIT in a manner similar to KIT mAb staining, and was trafficked intracellularly in vitro The KIT aptamer bound dissociated primary human GIST cells in a mutation agnostic manner such that tumors with KIT and PDGFRA mutations were labeled. In addition, the KIT aptamer specifically labeled intact human GIST tissue ex vivo, as well as peritoneal xenografts in mice with high sensitivity. These results represent the first use of an aptamer-based method for targeted detection of GIST in vitro and in vivo.
Insights
Researchers developed a novel DNA aptamer to specifically target and label gastrointestinal stromal tumors (GIST). This aptamer binds to KIT protein, enabling precise detection of GIST cells and tissues both in vitro and in vivo.
Area of Science:
- Biotechnology
- Oncology
- Molecular Biology
Background:
- Gastrointestinal stromal tumor (GIST) is the most common sarcoma, often driven by KIT mutations.
- Targeted KIT inhibition has advanced GIST therapy and precision medicine.
- There is a need for novel diagnostic and targeting tools for GIST.
Purpose of the Study:
- To investigate the utility of a KIT-specific DNA aptamer for targeted GIST detection.
- To evaluate the aptamer's specificity and efficacy for labeling GIST cells and tissues.
Main Methods:
- Development and characterization of a DNA aptamer targeting the KIT protein.
- In vitro studies assessing aptamer binding to GIST cells.
- In vivo studies evaluating aptamer labeling of human GIST xenografts in mice.
Main Results:
- The anti-KIT DNA aptamer demonstrated specific and KIT-dependent binding to GIST cells in vitro.
- The aptamer effectively labeled primary human GIST cells, irrespective of KIT or PDGFRA mutation status.
- High sensitivity labeling of intact human GIST tissue ex vivo and GIST xenografts in vivo was achieved.
Conclusions:
- A KIT-specific DNA aptamer can be used for targeted GIST cell labeling.
- This aptamer shows promise for mutation-agnostic detection of GIST in vitro and in vivo.
- Aptamer-based methods offer a novel approach for GIST detection and potentially targeted therapy.
