Aptamers as targeted therapeutics: current potential and challenges.
Nucleic acid aptamers, often termed 'chemical antibodies', are functionally comparable to traditional antibodies, but offer several advantages, including their relatively small physical size, flexible structure, quick chemical production, versatile chemical modification, high stability and lack of immunogenicity. In addition, many aptamers are internalized upon binding to cellular receptors, making them useful targeted delivery agents for small interfering RNAs (siRNAs), microRNAs and conventional drugs. However, several crucial factors have delayed the clinical translation of therapeutic aptamers, such as their inherent physicochemical characteristics and lack of safety data. This Review discusses these challenges, highlighting recent clinical developments and technological advances that have revived the impetus for this promising class of therapeutics.
Department of Molecular and Cellular Biology, Beckman Research Institute of City of Hope, 1500 E. Duarte Rd, Duarte, California 91010, USA.
Jiehua
J
Zhou
Department of Molecular and Cellular Biology, Beckman Research Institute of City of Hope, 1500 E. Duarte Rd, Duarte, California 91010, USA.|Irell and Manella Graduate School of Biological Sciences, Beckman Research Institute of City of Hope, 1500 E. Duarte Rd, Duarte, California 91010, USA.
John
J
Rossi
D000818:Animals; D052157:Aptamers, Nucleotide / Q000235:genetics* / Q000378:metabolism; D016503:Drug Delivery Systems / Q000379:methods / Q000639:trends*; D006801:Humans; D009696:Nucleic Acids / Q000235:genetics* / Q000378:metabolism; D034741:RNA, Small Interfering / Q000235:genetics / Q000378:metabolism; D052156:SELEX Aptamer Technique / Q000379:methods / Q000639:trends*
D052157:Aptamers, Nucleotide; D009696:Nucleic Acids; D034741:RNA, Small Interfering