Drug name: shRNA

Related CSCTT Targets

KIT (SCFR) [ref.1]P10721
HOTAIR [ref.2]
SMO [ref.3]Q99835
EZH2 [ref.4]Q15910
Aurora A [ref.5]O14965
Telomerase [ref.6]O14746
ROCK1 [ref.7]P70335
PKCδ [ref.8]Q05655
STAT3 [ref.6,9]P40763
GD3S [ref.10]Q92185
Notch1 [ref.11,12]P46531
Notch4 [ref.11]Q99466
Pyk2 [ref.13]Q14289
L1CAM [ref.14]P32004
c-Myc [ref.15]P01106
BMI1 [ref.16]P35226
ZEB1 [ref.17]P37275
ZEB2 [ref.17]O60315
Chk1 [ref.18]O14757
RAF1 [ref.19]P04049
CTGF [ref.20]P29279
uPAR [ref.21]Q03405
Ubiquitin Ligases [ref.22]


A small hairpin RNA or short hairpin RNA (shRNA) is a sequence of RNA that makes a tight hairpin turn that can be used to silence target gene expression via RNA interference (RNAi). Expression of shRNA in cells is typically accomplished by delivery of plasmids or through viral or bacterial vectors. The promoter choice is essential to achieve robust shRNA expression. At first, polymerase III promoters such as U6 and H1 were used; however, these promoters lack spatial and temporal control. As such, there has been a shift to using polymerase II promoters to regulate expression of shRNA. shRNA is an advantageous mediator of RNAi in that it has a relatively low rate of degradation and turnover.

Expression of shRNA in cells can be obtained by delivery of plasmids or through viral or bacterial vectors. Through transfection vectors to cells to obtain shRNA expression can be accomplished using commercially available reagents in vitro, but not in vivo and limited the utility.

Several challenges typically confront shRNA-based therapeutics. The most significant challenge is delivery. shRNA is typically delivered through use of a vector, and although they are generally efficient, they pose significant safety concerns. In particular, viral based gene therapy approaches have proved dangerous in past clinical trials. Potential oversaturation of RISC is also a problem. If the shRNA is expressed at levels that are too high the cell might not be able to correctly process the endogenous RNA which could cause significant problems. Another challenge is the possibility that the patient might mount an immune response against the therapy. Finally, there might be off-target effects and the shRNA could silence other unintended genes. In developing successful new shRNA-based therapeutics, all of these challenges must be taken into account.




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    Dou, J., et al. (2016). Am J Transl Res 8(1): 98-108. [ 27069543 ]
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    Chung, S. S., et al. (2013). PLoS One 8(12): e83971. [ 24386318 ]
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    Wang, J., et al. (2008). PLoS One 3(11): e3769. [ 19020659 ]
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    Chiba, T., et al. (2008). Cancer Res 68(19): 7742-7749. [ 18829528 ]
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    Wellner, U., et al. (2009). Nat Cell Biol 11(12): 1487-1495. [ 19935649 ]
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    Wang, X., et al. (2012). Oncol Rep 28(6): 2247-2254. [ 23027394 ]
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    Chang, C. J., et al. (2011). Cancer Cell 19(1): 86-100. [ 21215703 ]
  • [20] MicroRNA-145 is downregulated in glial tumors and regulates glioma cell migration by targeting connective tissue growth factor.
    Lee, H. K., et al. (2013). PLoS One 8(2): e54652. [ 23390502 ]
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    Asuthkar, S., et al. (2012). J Biol Chem 287(24): 20576-20589. [ 22511755 ]
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    Low, J., et al. (2012). J Biomol Screen 17(2): 152-162. [ 21956171 ]

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