Therapeutic potential of RNAi in metabolic diseases.
Over the past years RNA interference (RNAi) has exploded as a new approach to manipulate gene expression in mammalian systems. More recently, RNAi has acquired interest as a potential therapeutic strategy. This review focuses on the potential therapeutic use of RNAi for metabolic diseases, the current understanding of RNAi biology, and how RNAi has been utilized to study the role of different genes in the pathogenesis of diabetes and obesity. Also reviewed are the in vivo proof-of-principle experiments that provide the preclinical justification for the development of RNAi-based therapeutics for diabetes and the key challenges that currently limit its application in the clinical setting.
Rondinone
Cristina M
CM
Department Metabolic Diseases, Hoffmann-La Roche, Nutley, NJ 07110, USA. cristina.rondinone@roche.com
D018390:Gene Targeting / Q000379:methods* / Q000639:trends; D005818:Genetic Engineering / Q000379:methods / Q000639:trends; D015316:Genetic Therapy / Q000379:methods* / Q000639:trends; D006801:Humans; D008659:Metabolic Diseases / Q000235:genetics* / Q000628:therapy*; D034622:RNA Interference; D034741:RNA, Small Interfering / Q000235:genetics*
D034741:RNA, Small Interfering