Engineering RNA for targeted siRNA delivery and medical application.
RNA engineering for nanotechnology and medical applications is an exciting emerging research field. RNA has intrinsically defined features on the nanometre scale and is a particularly interesting candidate for such applications due to its amazing diversity, flexibility and versatility in structure and function. Specifically, the current use of siRNA to silence target genes involved in disease has generated much excitement in the scientific community. The intrinsic ability to sequence-specifically downregulate gene expression in a temporally- and spatially controlled fashion has led to heightened interest and rapid development of siRNA-based therapeutics. Although methods for gene silencing have been achieved with high efficacy and specificity in vitro, the effective delivery of nucleic acids to specific cells in vivo has been a hurdle for RNA therapeutics. This article covers different RNA-based approaches for diagnosis, prevention and treatment of human disease, with a focus on the latest developments of non-viral carriers of siRNA for delivery in vivo. The applications and challenges of siRNA therapy, as well as potential solutions to these problems, the approaches for using phi29 pRNA-based vectors as polyvalent vehicles for specific delivery of siRNA, ribozymes, drugs or other therapeutic agents to specific cells for therapy will also be addressed.
Department of Biomedical Engineering College of Engineering/College of Medicine, University of Cincinnati, Cincinnati, OH 45221, USA. guop@purdue.edu
Peixuan
P
Guo
Oana
O
Coban
Nicholas M
NM
Snead
Joe
J
Trebley
Steve
S
Hoeprich
Songchuan
S
Guo
Yi
Y
Shu
D000818:Animals; D052157:Aptamers, Nucleotide / Q000627:therapeutic use; D050091:Dendrimers / Q000378:metabolism / Q000627:therapeutic use; D004337:Drug Carriers / Q000378:metabolism / Q000627:therapeutic use; D016503:Drug Delivery Systems; D004355:Drug Stability; D018390:Gene Targeting; D015316:Genetic Therapy; D006801:Humans; D008081:Liposomes / Q000378:metabolism / Q000627:therapeutic use; D051379:Mice; D008968:Molecular Conformation; D012313:RNA / Q000235:genetics / Q000378:metabolism / Q000627:therapeutic use*; D034741:RNA, Small Interfering / Q000009:adverse effects / Q000378:metabolism / Q000493:pharmacokinetics / Q000627:therapeutic use*; D052156:SELEX Aptamer Technique
D052157:Aptamers, Nucleotide; D050091:Dendrimers; D004337:Drug Carriers; D008081:Liposomes; D034741:RNA, Small Interfering; D012313:RNA