Scientific Publications by FDA Staff

DNA RNA Nanotechnol 2016 Jan;2(1):42-52

Aptamer guided delivery of nucleic acid-based nanoparticles.

Panigaj M, Reiser J

Targeted delivery of bioactive compounds is a key part of successful therapies. In this context, nucleic acid and protein-based aptamers have been shown to bind therapeutically relevant targets including receptors. In the last decade, nucleic acid-based therapeutics coupled to aptamers have emerged as a viable strategy for cell specific delivery. Additionally, recent developments in nucleic acid nanotechnology offer an abundance of possibilities to rationally design aptamer targeted RNA or DNA nanoparticles involving combinatorial use of various intrinsic functionalities. Although a host of issues including stability, safety and intracellular trafficking remain to be addressed, aptamers as simple functional chimeras or as parts of multifunctional self-assembled RNA/DNA nanostructures hold great potential for clinical applications.