Aptamers as promising molecular recognition elements for diagnostics and therapeutics in the central nervous system
Oligonucleotide aptamers are short, synthetic, single-stranded DNA or RNA able to recognize and bind to a multitude of targets ranging from small molecules to cells. Aptamers have emerged as valuable tools for fundamental research, clinical diagnosis, and therapy. Due to their small size, strong target affinity, lack of immunogenicity, and ease of chemical modification, aptamers are an attractive alternative to other molecular recognition elements, such as antibodies. Although it is a challenging environment, the central nervous system and related molecular targets present an exciting potential area for aptamer research. Aptamers hold promise for targeted drug delivery, diagnostics, and therapeutics. Here we review recent advances in aptamer research for neurotransmitter and neurotoxin targets, demyelinating disease and spinal cord injury, cerebrovascular disorders, pathologies related to protein aggregation (Alzheimer's, Parkinson's, and prions), brain cancer (glioblastomas and gliomas), and regulation of receptor function. Challenges and limitations posed by the blood brain barrier are described. Future perspectives for the application of aptamers to the central nervous system are also discussed.
|Journal||Nucleic Acid Therapeutics|
McConnell, E.M. (Erin M.), Holahan, M.R, & DeRosa, M.C. (2014). Aptamers as promising molecular recognition elements for diagnostics and therapeutics in the central nervous system. Nucleic Acid Therapeutics, 24(6), 388–404. doi:10.1089/nat.2014.0492