Aptamer-based neuroinflammation target discovery and targeted drug delivery platform

Overview

This research program focuses on discovering novel biomarkers associated with neuroinflammation and developing targeted drug delivery systems using aptamer technology. Neuroinflammation plays a central role in neurodegenerative disorders including Alzheimer's and Parkinson's disease, where inflammatory neuronal cells display distinct biomarker profiles that could enable earlier diagnosis and more precise therapeutic intervention. By applying systematic evolution of ligands by exponential enrichment (SELEX), the platform aims to generate aptamers with high specificity for inflammatory neuronal cells, even without prior knowledge of the target. This opens a pathway to uncover previously unknown disease-associated biomarkers and to deliver therapeutics directly to affected cells, potentially improving treatment outcomes while reducing required drug dosages.

Technical specifications

Core capabilities:

• Cell-SELEX selection against LPS-induced human microglial HMC3 cells and rat pheochromocytoma PC12 cell lines, with untreated cells used for negative SELEX to ensure specificity
• Binding characterization using flow cytometry and imaging to evaluate affinity and specificity of enriched aptamer pools
• Target identification through biotin pull-down assays coupled with mass spectrometry to reveal novel neuroinflammation-associated biomarkers
• Tissue validation via imaging in brain tissues of LPS-induced rat models to confirm aptamer binding to inflammatory brain tissue
• Aptamer-based biosensor development capable of detecting targets that lack suitable antibodies or existing binding ligands
• Targeted drug delivery demonstrated previously with corneal aptamers achieving nanomolar binding affinity and prolonged drug retention up to 24 hours in vivo

The aptamer platform offers advantages over traditional antibody-based approaches, including the ability to select binders without prior target knowledge, chemical synthesis and modification flexibility, and applicability across biosensing, biomarker discovery, and therapeutic delivery.

Technology readiness level

The SELEX methodology has been validated through prior work demonstrating successful aptamer selection with nanomolar binding affinity for corneal targets and effective in vivo drug delivery in rabbit eyes with sustained retention. A published review documents diagnostic and therapeutic aptamers for inflammatory biomolecules in neurodegenerative contexts. The current program is at the preclinical validation stage, with planned in vitro characterization of aptamer binding affinity, target identification via mass spectrometry, and in vivo validation in LPS-induced rat brain tissue. Upon demonstrating specificity to inflammatory brain tissue, the next milestone is development of an aptamer-based targeted drug delivery system for neuroinflammatory disorders.