Challenges for RNAi in vivo.
Synthetic small interfering RNA (siRNA) has become a valuable tool for investigating gene function in cell culture. This success has led to high expectations for siRNA as a tool for in vivo investigation and as a platform for therapeutic development. siRNA in cell culture owes much of its success to years of development of traditional antisense oligonucleotides, and in vivo applications will also benefit from previous experience in this regard. However, the duplex nature of siRNA presents significant obstacles that will need to be overcome. Here, we discuss the current status of in vivo siRNA technology and describe some of the barriers to widespread application of RNAi-mediated gene silencing in mammals.
Departments of Pharmacology and Biochemistry, University of Texas Southwestern Medical Center, Dallas, TX 75390-9041, USA.
Zain
Z
Paroo
David R
DR
Corey
D000818:Animals; D002478:Cells, Cultured; D020868:Gene Silencing; D006801:Humans; D016376:Oligonucleotides, Antisense / Q000737:chemistry*; D010710:Phosphates / Q000737:chemistry*; D034741:RNA, Small Interfering / Q000737:chemistry / Q000493:pharmacokinetics*; D014162:Transfection
D016376:Oligonucleotides, Antisense; D010710:Phosphates; D034741:RNA, Small Interfering; C083120:phosphorodithioic acid