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Often described as "chemical antibodies", aptamers are short oligonucleotides of DNA/RNA that fold into defined tertiary structures and bind to specific targets with high affinity and specificity. Through a process called systematic evolution of ligands by exponential enrichment (SELEX), a number of aptamers have been identified against various targets including organic compounds, nucleotides, proteins and even whole cells and organisms.
RNA aptamers can directly block or interrupt the functions of target proteins making them promising therapeutic agents for the treatment of human maladies. To date, the FDA has approved one RNA aptamer drug (Fig1: Macugen) for the treatment of age-related macular degeneration (AMD). Ten aptamer drugs have undergone clinical trials for the treatment of various conditions, including coagulation, oncology, and inflammation.
Fig1. Macugen, the first FDA approved aptamer-based drug.
Additionally, RNA aptamers also have been developed for various applications such as diagnostics, molecular imaging, biomarker discovery, target validation, disease cell detection and drug delivery. For example, various cell type-specific aptamers have been conjugated with therapeutic agents (siRNA, microRNA, anti-miR, therapeutic aptamer, or toxins), or delivery vehicles (organic or inorganic nanocarriers) for targeted delivery in a cell type-specific manner. By taking advantage of the high affinity and specificity of aptamers, therapeutic compounds can be targeted to the desired cells or tissues, thereby improving their local concentration and therapeutic efficacy.
IntegrateRNA has many years of experience in RNA research, providing professional and high quality services and products for RNA basic research, drug discovery and clinical research. Our custom RNA aptamer services including aptamer synthesis, library screening, sequence analysis, characterization and optimization. If you have any question, please feel free to contact us.