Aptamers: Potential Diagnostic and Therapeutic Agents for Blood Diseases

Aptamers are RNA/DNA oligonucleotide molecules that specifically bind to a targeted complementary molecule. As potential recognition elements with promising diagnostic and therapeutic applications, aptamers, such as monoclonal antibodies, could provide many treatment and diagnostic options for blood diseases. Aptamers present several superior features over antibodies, including a simple in vitro selection and production, ease of modification and conjugation, high stability, and low immunogenicity. Emerging as promising alternatives to antibodies, aptamers could overcome the present limitations of monoclonal antibody therapy to provide novel diagnostic, therapeutic, and preventive treatments for blood diseases. Researchers in several biomedical areas, such as biomarker detection, diagnosis, imaging, and targeted therapy, have widely investigated aptamers, and several aptamers have been developed over the past two decades. One of these is the pegaptanib sodium injection, an aptamer-based therapeutic that functions as an anti-angiogenic medicine, and it is the first aptamer approved by the U.S. Food and Drug Administration (FDA) for therapeutic use. Several other aptamers are now in clinical trials. In this review, we highlight the current state of aptamers in the clinical trial program and introduce some promising aptamers currently in pre-clinical development for blood diseases.

Automated summary

Aptamers are RNA/DNA oligonucleotide molecules that specifically bind to targeted molecules. They have potential diagnostic and therapeutic applications for blood diseases and offer advantages over antibodies in terms of selection, production, modification, stability, and immunogenicity. Several aptamers have been developed and investigated in various biomedical areas. The pegaptanib sodium injection, an aptamer-based therapeutic, has been approved by the FDA for therapeutic use. Other aptamers are also in clinical trials. This review highlights the current status and promising aptamers in pre-clinical development for blood diseases.