Delivery of RNA interference

被引:164
作者
Li, Charles X. [1 ]
Parker, Amy [1 ]
Menocal, Ellen [1 ]
Xiang, Shuanglin [1 ]
Borodyansky, Laura [1 ]
Fruehauf, Johannes H. [1 ]
机构
[1] Harvard Univ, Sch Med, Div Gastroenterol, Beth Israel Deaconess Med Ctr, Boston, MA 02215 USA
关键词
RNA interference; gene therapy; gene delivery; therapeutic RNAi; transkingdom RNA interference;
D O I
10.4161/cc.5.18.3192
中图分类号
Q2 [细胞生物学];
学科分类号
071009 ; 090102 ;
摘要
Over the last few years, RNA Interference (RNAi), a naturally occurring mechanism of gene regulation conserved in plant and mammalian cells, has opened numerous novel opportunities for basic research across the field of biology. While RNAi has helped accelerate discovery and understanding of gene functions, it also has great potential as a therapeutic and potentially prophylactic modality. Challenging diseases failing conventional therapeutics could become treatable by specific silencing of key pathogenic genes. More specifically, therapeutic targets previously deemed "undruggable" by small molecules, are now coming within reach of RNAi based therapy. For RNAi to be effective and elicit gene silencing response, the double-stranded RNA molecules must be delivered to the target cell. Unfortunately, delivery of these RNA duplexes has been challenging, halting rapid development of RNAi-based therapies. In this review we present current advancements in the field of siRNA delivery methods, including the pros and cons of each method.
引用
收藏
页码:2103 / 2109
页数:7
相关论文
共 59 条
[21]   American Society of Gene Therapy (ASGT) ad hoc subcommittee on retroviral-mediated gene transfer to hematopoietic stem cells [J].
Kohn, DB ;
Sadelain, M ;
Dunbar, C ;
Bodine, D ;
Kiem, HP ;
Candotti, F ;
Tisdale, J ;
Riviére, I ;
Blau, CA ;
Richard, RE ;
Sorrentino, B ;
Nolta, J ;
Malech, H ;
Brenner, M ;
Cornetta, K ;
Cavagnaro, J ;
High, K ;
Glorioso, J .
MOLECULAR THERAPY, 2003, 8 (02) :180-187
[22]   PGC-1 promotes insulin resistance in liver through PPAR-α-dependent induction of TRB-3 [J].
Koo, SH ;
Satoh, H ;
Herzig, S ;
Lee, CH ;
Hedrick, S ;
Kulkarni, R ;
Evans, RM ;
Olefsky, J ;
Montminy, M .
NATURE MEDICINE, 2004, 10 (05) :530-534
[23]   Therapeutic EphA2 gene targeting in vivo using neutral liposomal small interfering RNA delivery [J].
Landen, CN ;
Chavez-Reyes, A ;
Bucana, C ;
Schmandt, R ;
Deavers, MT ;
Lopez-Berestein, G ;
Sood, AK .
CANCER RESEARCH, 2005, 65 (15) :6910-6918
[24]   In vivo activity of nuclease-resistant siRNAs [J].
Layzer, JM ;
McCaffrey, AP ;
Tanner, AK ;
Huang, Z ;
Kay, MA ;
Sullenger, BA .
RNA, 2004, 10 (05) :766-771
[25]   Efficient delivery of siRNA for inhibition of gene expression in postnatal mice [J].
Lewis, DL ;
Hagstrom, JE ;
Loomis, AG ;
Wolff, JA ;
Herweijer, H .
NATURE GENETICS, 2002, 32 (01) :107-108
[26]   Use of adenovirus-delivered siRNA to target oncoprotein p28GANK in hepatocellular carcinoma [J].
Li, HH ;
Fu, XY ;
Chen, Y ;
Hong, Y ;
Tan, YX ;
Cao, HF ;
Wu, MC ;
Wang, HY .
GASTROENTEROLOGY, 2005, 128 (07) :2029-2041
[27]   Hydrodynamics-based transfection in animals by systemic administration of plasmid DNA [J].
Liu, F ;
Song, YK ;
Liu, D .
GENE THERAPY, 1999, 6 (07) :1258-1266
[28]   Efficient transduction of liver and muscle after in utero injection of lentiviral vectors with different pseudotypes [J].
MacKenzie, TC ;
Kobinger, GP ;
Kootstra, NA ;
Radu, A ;
Sena-Esteves, M ;
Bouchard, S ;
Wilson, JM ;
Verma, IM ;
Flake, AW .
MOLECULAR THERAPY, 2002, 6 (03) :349-358
[29]   Gene expression - RNA interference in adult mice [J].
McCaffrey, AP ;
Meuse, L ;
Pham, TTT ;
Conklin, DS ;
Hannon, GJ ;
Kay, MA .
NATURE, 2002, 418 (6893) :38-39
[30]   siRNA-induced caveolin-1 knockdown in mice increases lung vascular permeability via the junctional pathway [J].
Miyawaki-Shimizu, K ;
Predescu, D ;
Shimizu, J ;
Broman, M ;
Predescu, S ;
Malik, AB .
AMERICAN JOURNAL OF PHYSIOLOGY-LUNG CELLULAR AND MOLECULAR PHYSIOLOGY, 2006, 290 (02) :L405-L413