title: Therapeutic potentials of gene silencing by RNA interference: principles, challenges, and new strategies.
abstract: During recent decades there have been remarkable advances in biology, in which one of the most important discoveries is RNA interference (RNAi). RNAi is a specific post-transcriptional regulatory pathway that can result in silencing gene functions. Efforts have been done to translate this new discovery into clinical applications for disease treatment. However, technical difficulties restrict the development of RNAi, including stability, off-target effects, immunostimulation and delivery problems. Researchers have attempted to surmount these barriers and improve the bioavailability and safety of RNAi-based therapeutics by optimizing the chemistry and structure of these molecules. This paper aimed to describe the principles of RNA interference, review the therapeutic potential in various diseases and discuss the new strategies for in vivo delivery of RNAi to overcome the challenges.
authors.affiliation: Department of Obstetrics & Gynaecology, Faculty of Medicine, The Chinese University of Hong Kong, Shatin, New Territories, Hong Kong, China; Shenzhen Research Institute, The Chinese University of Hong Kong, Hong Kong, China.
authors.forename: Yan
authors.initials: Y
authors.lastname: Deng
authors.affiliation: Department of Obstetrics & Gynaecology, Faculty of Medicine, The Chinese University of Hong Kong, Shatin, New Territories, Hong Kong, China.
authors.forename: Chi Chiu
authors.initials: CC
authors.lastname: Wang
authors.affiliation: Department of Obstetrics & Gynaecology, Faculty of Medicine, The Chinese University of Hong Kong, Shatin, New Territories, Hong Kong, China; Shenzhen Research Institute, The Chinese University of Hong Kong, Hong Kong, China.
authors.forename: Kwong Wai
authors.initials: KW
authors.lastname: Choy
authors.affiliation: State Key Laboratory of Natural and Biomimetic Drugs, School of Pharmaceutical Sciences, Peking University, Beijing, China.
authors.forename: Quan
authors.initials: Q
authors.lastname: Du
authors.affiliation: Department of Obstetrics & Gynaecology, Faculty of Medicine, The Chinese University of Hong Kong, Shatin, New Territories, Hong Kong, China.
authors.forename: Jiao
authors.initials: J
authors.lastname: Chen
authors.affiliation: Linyi City People's Hospital Division Medical District Nursing Office, Linyi, China.
authors.forename: Qin
authors.initials: Q
authors.lastname: Wang
authors.affiliation: Department of Obstetrics & Gynaecology, Faculty of Medicine, The Chinese University of Hong Kong, Shatin, New Territories, Hong Kong, China.
authors.forename: Lu
authors.initials: L
authors.lastname: Li
authors.affiliation: Department of Obstetrics & Gynaecology, Faculty of Medicine, The Chinese University of Hong Kong, Shatin, New Territories, Hong Kong, China.
authors.forename: Tony Kwok Hung
authors.initials: TK
authors.lastname: Chung
authors.affiliation: Department of Obstetrics & Gynaecology, Faculty of Medicine, The Chinese University of Hong Kong, Shatin, New Territories, Hong Kong, China; Shenzhen Research Institute, The Chinese University of Hong Kong, Hong Kong, China. Electronic address: tangtao@cuhk.edu.hk.
authors.forename: Tao
authors.initials: T
authors.lastname: Tang
mesh_terms: D000818:Animals; D002318:Cardiovascular Diseases / Q000235:genetics / Q000628:therapy; D016503:Drug Delivery Systems; D020868:Gene Silencing; D015316:Genetic Therapy / Q000009:adverse effects / Q000379:methods* / Q000639:trends; D005822:Genetic Vectors; D015658:HIV Infections / Q000235:genetics / Q000628:therapy; D019698:Hepatitis C, Chronic / Q000235:genetics / Q000628:therapy; D006801:Humans; D009369:Neoplasms / Q000235:genetics / Q000628:therapy; D019636:Neurodegenerative Diseases / Q000235:genetics / Q000628:therapy; D034622:RNA Interference / Q000276:immunology; D020871:RNA Stability; D034741:RNA, Small Interfering / Q000008:administration & dosage / Q000235:genetics / Q000627:therapeutic use
chemical_list: D034741:RNA, Small Interfering
keywords: Gene-silencing; RNA interference; RNAi delivery; Therapeutics