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| 1 | RNAi mechanisms in Huntington’s disease therapy:siRNA versus shRNA显示文摘Huntington’s Disease(HD)is a genetically dominant trinucleotide repeat disorder resulting from CAG repeats within the Huntingtin(HTT)gene exceeding a normal range(>36 CAGs).Symptoms of the disease manifest in middle age and include chorea,dystonia,and cognitive decline.Typical latency from diagnosis to death is 20 years.There are currently no disease-modifying therapies available to HD patients.RNAi is a potentially curative therapy for HD.A popular line of research employs siRNA or antisense oligonucleotides(ASO)to knock down mutant Huntingtin mRNA(mHTT).Unfortunately,this modality requires repeated dosing,commonly exhibit off target effects(OTEs),and exert renal and hepatic toxicity.In contrast,a single AAV-mediated short-hairpin RNA(shRNA)dose can last years with low toxicity.In addition,we highlight research indicating that shRNA elicits fewer OTEs than siRNA when tested head-tohead.Despite this promise,shRNA therapy has been held back by difficulties controlling expression(oversaturating cells with toxic levels of RNA construct).In this review,we compare RNAi modalities for HD and propose novel methods of optimizing shRNA expression and on-target fidelity. | Sebastian Aguiar Bram van der Gaag Francesco Albert Bosco Cortese | 2017 | Translational Neurodegeneration2017，6，1： | 3 |
| 2 | Endoscopic and Percutaneous Preoperative Biliary Drainage in Patients with Suspected Hilar Cholangiocarcinoma显示文摘 | Jaap J. Kloek Niels A. Gaag Yalda Aziz Erik A. J. Rauws Otto M. Delden Johan S. Lameris Olivier R. C. Busch Dirk J. Gouma Thomas M. Gulik | 2010 | Journal of Gastrointestinal Surgery2010，，1： | 3 |
| 3 | Advances in on-line drinking water quality monitoring and early warning systems显示文摘 | Michael V. Storey Bram van der Gaag Brendan P. Burns | 2010 | Water Research2010，，2： | 2 |
| 4 | Education and Earnings in the People's Republic of China 显示文摘 | Jamison D van der Gaag J | | Economics of Education Review0，6，： | 1 |
| 5 | A quantitative assay for the number of routine lymphoma cells capable of proliferation in vivo显示文摘 | Bruce WR Gaag HVD | 1963 | Nature1963，199，： | 1 |
| 6 | Conduciveness of different soilless growing media to Pythium root and crown rot of cucumber under near-commercial conditions显示文摘 | van der Gaag DJ Wever G | 2005 | European Journal of Plant Pathology2005，112，1： | 1 |
| 7 | Aquantitative assay for the number of murine lymphoma cells capable of proliferationin vivo 显示文摘 | Bruce WR Van Der Gaag H | 1963 | Nature1963，199，： | 1 |
| 8 | Clinical presentation of Graves ' ophthalmopathy 显示文摘 | WIERSINGA W M SMIT T VAN DER GAAG R | 1989 | Ophthalmic Res1989，21，2： | 1 |
| 9 | Effect of consumption of red wine,spirits,and beer on serum homocysteine显示文摘 | van der Gaag MS Ubbink JB Sillanaukee P | 2000 | Lancet2000，355，9214： | 1 |
| 10 | How effective is intermittent chlorination to control adult mussel fouling in cooling water systems? 显示文摘 | Raiagopal S Velde G Gaag M | 2003 | Water Research2003，37，2： | 1 |
| 11 | A quantitative assay for the number of murine lymphoma cells capable of proliferation in vivo 显示文摘 | BRUCE WR VAN DER GAAG H | 1963 | Nature1963，199，： | 1 |
| 12 | 查看详情显示文摘 | Jansen J C van der Gaag F J van Bekkum H | | 0，，： | 1 |
| 13 | Suppressiveness of 18 composts against 7 pathosystems: variability in pathogen response显示文摘 | Termorshuizen A J van Rijn E van der Gaag D J | 2006 | Soil Biology and Biochemistry2006，38，8： | 1 |
| 14 | High expression of the bile salt-homeostatic hormone fibroblast growth factor 19 in the liver of patients with extrahepatic cholestasis显示文摘 | Schaap FG van der Gaag NA Gouma D J | 2009 | Hepatology2009，49，4： | 1 |
| 15 | Bayesian belief networks:Odds and ends显示文摘 | Gaag L C V | 1996 | The Computer Journal1996，39，2： | 1 |
| 16 | Clinical criteria for the assessment of disease activity in Graves’ophthalmopathy:a novel approach显示文摘 | Mourits MP Koornneef L Wiersinga WM Prummel MF Berg-hout A van der Gaag R | 1989 | Br J Ophthalmol1989，73，8： | 1 |
| 17 | Daily moderate alcohol consumption increases serum paraoxonase activity: a diet-controlled,randomized intervention study in middle-aged men显示文摘 | Vander Gaag MS Van Toi A Scheek LM | 1999 | Atherosclerosis1999，147，： | 1 |
| 18 | Corre- lates and consequences of internalized stigma for people living with mentat illness:A systematic review and meta-analysis显示文摘 | Staring ABP van der Gaag M van den Berger M | 2010 | Social Science & Medicine2010，71，12： | 1 |
| 19 | Functional and medical outcomes after tailored surgery for pain due to chronic pancreatitis显示文摘 | Van der Gaag NA Van Gulik TM Busch OR | | 0，，02： | 1 |
| 20 | Treating trauma in psychosis with EMDR: A pilot study显示文摘 | David P.G. van den Berg Mark van der Gaag | 2011 | Journal of Behavior Therapy and Experimental Psychiatry2011，，1： | 1 |
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