title: Noncoding oligonucleotides: the belle of the ball in gene therapy.
abstract: Gene therapy carries the promise of cures for many diseases based on manipulating the expression of a person's genes toward the therapeutic goal. The relevance of noncoding oligonucleotides to human disease is attracting widespread attention. Noncoding oligonucleotides are not only involved in gene regulation, but can also be modified into therapeutic tools. There are many strategies that leverage noncoding oligonucleotides for gene therapy, including small interfering RNAs, antisense oligonucleotides, aptamers, ribozymes, decoys, and bacteriophage phi 29 RNAs. In this chapter, we will provide a broad, comprehensive overview of gene therapies that use noncoding oligonucleotides for disease treatment. The mechanism and development of each therapeutic will be described, with a particular focus on its clinical development. Finally, we will discuss the challenges associated with developing nucleic acid therapeutics and the prospects for future success.
authors.affiliation: Department of Molecular and Cellular Biology, Beckman Research Institute of City of Hope, Duarte, CA, USA.
authors.forename: Ka-To
authors.initials: KT
authors.lastname: Shum
authors.affiliation: Department of Molecular and Cellular Biology, Beckman Research Institute of City of Hope, Duarte, CA, USA.
authors.forename: John J
authors.initials: JJ
authors.lastname: Rossi
mesh_terms: D002986:Clinical Trials as Topic; D015316:Genetic Therapy; D005822:Genetic Vectors; D006801:Humans; D009841:Oligonucleotides / Q000627:therapeutic use*; D022661:RNA, Untranslated / Q000627:therapeutic use*
chemical_list: D009841:Oligonucleotides; D022661:RNA, Untranslated
keywords: Antisense; Aptamer; Bacteriophage phi29; Decoy; Gene therapy; RNA; RNAi; Ribozyme