Aptamer-Based Targeted Cancer Therapy: From Target Discovery to Therapeutic Application

Despite remarkable progress in precision oncology, achieving highly selective and efficient delivery of therapeutic agents to tumor tissues remains a fundamental challenge in cancer treatment. Aptamers — single-stranded DNA or RNA oligonucleotides selected through Systematic Evolution of Ligands by EXponential enrichment (SELEX) — bind to specific molecular targets with high affinity and specificity comparable to that of monoclonal antibodies, yet offer distinct advantages including low immunogenicity, facile chemical synthesis, small molecular size, excellent tumor tissue penetration, and broad engineering flexibility.

Recent advances in aptamer–drug conjugates (ApDCs), aptamer-functionalized nanocarriers, nucleic acid therapeutics, and aptamer-guided gene editing have substantially expanded the translational potential of aptamer-based anti-cancer interventions. In parallel, the integration of artificial intelligence (AI) and machine learning (ML) technologies is accelerating aptamer discovery, sequence optimization, target identification, binding affinity prediction, and clinical pharmacology modeling — opening new avenues for next-generation precision therapeutics.

Critically, the pharmacological development of aptamer-based anti-cancer agents demands rigorous characterization of their pharmacokinetics (PK), pharmacodynamics (PD), biodistribution, metabolic stability, and safety profiles to support regulatory approval and clinical translation. These pharmacological dimensions are integral to the responsible advancement of aptamers from the bench to the bedside.

This Research Topic provides a comprehensive, multidisciplinary platform to advance aptamer-targeted cancer therapy — from molecular recognition and target discovery, through drug design and delivery system development, to preclinical pharmacology and clinical translation.

Subtopics:

• Aptamer Discovery for Cancer Targets

• AI-Driven Aptamer Discovery and Sequence Optimization

• Aptamer–Drug Conjugates (ApDCs)

• Aptamer-Functionalized Nanomedicine Platforms

• Aptamer-siRNA and RNA Therapeutics Delivery

• Aptamer-Guided Gene Editing in Cancer

• AI-Guided Clinical Translation of Aptamer Therapeutics

• Pharmacokinetics, Pharmacodynamics, and Safety Profiling of Aptamer Therapeutics

• Translational and Clinical Development of Aptamer Therapeutics