Data from Anti-KIT DNA Aptamer for Targeted Labeling of Gastrointestinal Stromal Tumor

Abstract

<div>Abstract<p>Gastrointestinal stromal tumor (GIST), the most common sarcoma, is characterized by KIT protein overexpression, and tumors are frequently driven by oncogenic <i>KIT</i> 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 <i>in vitro</i>. Functionally, the KIT aptamer bound extracellular KIT in a manner similar to KIT mAb staining, and was trafficked intracellularly <i>in vitro</i>. The KIT aptamer bound dissociated primary human GIST cells in a mutation agnostic manner such that tumors with <i>KIT</i> and <i>PDGFRA</i> mutations were labeled. In addition, the KIT aptamer specifically labeled intact human GIST tissue <i>ex vivo</i>, 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 <i>in vitro</i> and <i>in vivo</i>.</p></div>