title: RNA interference therapeutics for cancer: challenges and opportunities (review).
abstract: RNA interference (RNAi) is a sequence-specific, post-transcriptional gene             silencing mechanism in animals and plants, which is mediated by double-stranded             RNA (dsRNA). There has recently been an increasing interest in harnessing the             gene silencing activity of dsRNA to develop novel drugs for the treatment of various             diseases, such as cancer, neurological disorders, age-related macular degeneration             and viral infections. Small interfering RNA (siRNA)-based drugs have distinct             advantages over conventional small molecule or protein-based drugs, including             high specificity, higher potency and reduced toxicity. However, there are several             technical obstacles to overcome before siRNA-based drugs reach the clinic. Delivery             of siRNA to the target tissues and stability in the serum remain a major challenge             and are the main focus of current research and development efforts. This review             focused primarily on the progress made in developing RNAi as therapeutics for             cancer and the challenges associated with its clinical development. Use of ligands             recognizing cell-specific receptors to achieve tumor-specific delivery of siRNA,             methods for enhanced siRNA delivery, improving the bioavailability and pharmacokinetic             properties of siRNA and reducing the off-target effects and non-specific gene             silencing are discussed in the light of current evidence.
authors.affiliation: Department of Biotechnology, Ranbaxy Laboratories Limited, Gurgaon 122 015, Haryana, India. roop.singh@ranbaxy.com
authors.forename: Roop Singh
authors.initials: RS
authors.lastname: Bora
authors.forename: Dikshi
authors.initials: D
authors.lastname: Gupta
authors.forename: Trilochan Kanwaljit Singh
authors.initials: TK
authors.lastname: Mukkur
authors.forename: Kulvinder Singh
authors.initials: KS
authors.lastname: Saini
mesh_terms: D000818:Animals; D002986:Clinical Trials as Topic; D006801:Humans; D009369:Neoplasms / Q000235:genetics / Q000628:therapy*; D034622:RNA Interference; D034741:RNA, Small Interfering / Q000009:adverse effects / Q000493:pharmacokinetics / Q000627:therapeutic use*
chemical_list: D034741:RNA, Small Interfering