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Items: 1 to 20 of 121


Allele-selective inhibition of huntingtin expression by switching to an miRNA-like RNAi mechanism.

Hu J, Liu J, Corey DR.

Chem Biol. 2010 Nov 24;17(11):1183-8. doi: 10.1016/j.chembiol.2010.10.013.


Allele-selective inhibition of mutant huntingtin expression with antisense oligonucleotides targeting the expanded CAG repeat.

Gagnon KT, Pendergraff HM, Deleavey GF, Swayze EE, Potier P, Randolph J, Roesch EB, Chattopadhyaya J, Damha MJ, Bennett CF, Montaillier C, Lemaitre M, Corey DR.

Biochemistry. 2010 Nov 30;49(47):10166-78. doi: 10.1021/bi101208k. Epub 2010 Nov 8.


Alpha-synuclein suppression by targeted small interfering RNA in the primate substantia nigra.

McCormack AL, Mak SK, Henderson JM, Bumcrot D, Farrer MJ, Di Monte DA.

PLoS One. 2010 Aug 11;5(8):e12122. doi: 10.1371/journal.pone.0012122.


Mammalian microRNAs predominantly act to decrease target mRNA levels.

Guo H, Ingolia NT, Weissman JS, Bartel DP.

Nature. 2010 Aug 12;466(7308):835-40. doi: 10.1038/nature09267.


Molecular mechanisms and potential therapeutical targets in Huntington's disease.

Zuccato C, Valenza M, Cattaneo E.

Physiol Rev. 2010 Jul;90(3):905-81. doi: 10.1152/physrev.00041.2009. Review.


RNAi-based treatment for neovascular age-related macular degeneration by Sirna-027.

Kaiser PK, Symons RC, Shah SM, Quinlan EJ, Tabandeh H, Do DV, Reisen G, Lockridge JA, Short B, Guerciolini R, Nguyen QD; Sirna-027 Study Investigators.

Am J Ophthalmol. 2010 Jul;150(1):33-39.e2. doi: 10.1016/j.ajo.2010.02.006.


Effect of mipomersen, an apolipoprotein B synthesis inhibitor, on low-density lipoprotein cholesterol in patients with familial hypercholesterolemia.

Akdim F, Visser ME, Tribble DL, Baker BF, Stroes ES, Yu R, Flaim JD, Su J, Stein EA, Kastelein JJ.

Am J Cardiol. 2010 May 15;105(10):1413-9. doi: 10.1016/j.amjcard.2010.01.003. Epub 2010 Mar 30.


A randomized, double-blind, placebo-controlled study of an RNAi-based therapy directed against respiratory syncytial virus.

DeVincenzo J, Lambkin-Williams R, Wilkinson T, Cehelsky J, Nochur S, Walsh E, Meyers R, Gollob J, Vaishnaw A.

Proc Natl Acad Sci U S A. 2010 May 11;107(19):8800-5. doi: 10.1073/pnas.0912186107. Epub 2010 Apr 26.


Aberrant Rab11-dependent trafficking of the neuronal glutamate transporter EAAC1 causes oxidative stress and cell death in Huntington's disease.

Li X, Valencia A, Sapp E, Masso N, Alexander J, Reeves P, Kegel KB, Aronin N, Difiglia M.

J Neurosci. 2010 Mar 31;30(13):4552-61. doi: 10.1523/JNEUROSCI.5865-09.2010.


RNAi therapeutics for CNS disorders.

Boudreau RL, Davidson BL.

Brain Res. 2010 Jun 18;1338:112-21. doi: 10.1016/j.brainres.2010.03.038. Epub 2010 Mar 20. Review.


Evidence of RNAi in humans from systemically administered siRNA via targeted nanoparticles.

Davis ME, Zuckerman JE, Choi CH, Seligson D, Tolcher A, Alabi CA, Yen Y, Heidel JD, Ribas A.

Nature. 2010 Apr 15;464(7291):1067-70. doi: 10.1038/nature08956. Epub 2010 Mar 21.


RNA targeting therapeutics: molecular mechanisms of antisense oligonucleotides as a therapeutic platform.

Bennett CF, Swayze EE.

Annu Rev Pharmacol Toxicol. 2010;50:259-93. doi: 10.1146/annurev.pharmtox.010909.105654. Review.


Treatment of malignant gliomas with TGF-beta2 antisense oligonucleotides.

Hau P, Jachimczak P, Bogdahn U.

Expert Rev Anticancer Ther. 2009 Nov;9(11):1663-74. doi: 10.1586/era.09.138. Review.


Safety and tolerability of putaminal AADC gene therapy for Parkinson disease.

Christine CW, Starr PA, Larson PS, Eberling JL, Jagust WJ, Hawkins RA, VanBrocklin HF, Wright JF, Bankiewicz KS, Aminoff MJ.

Neurology. 2009 Nov 17;73(20):1662-9. doi: 10.1212/WNL.0b013e3181c29356. Epub 2009 Oct 14.


Mutant huntingtin impairs vesicle formation from recycling endosomes by interfering with Rab11 activity.

Li X, Standley C, Sapp E, Valencia A, Qin ZH, Kegel KB, Yoder J, Comer-Tierney LA, Esteves M, Chase K, Alexander J, Masso N, Sobin L, Bellve K, Tuft R, Lifshitz L, Fogarty K, Aronin N, DiFiglia M.

Mol Cell Biol. 2009 Nov;29(22):6106-16. doi: 10.1128/MCB.00420-09. Epub 2009 Sep 14.


Image-guided convection-enhanced delivery of muscimol to the primate brain.

Heiss JD, Walbridge S, Asthagiri AR, Lonser RR.

J Neurosurg. 2010 Apr;112(4):790-5. doi: 10.3171/2009.7.JNS09652.


A phase I/II study of GTI-2040 plus docetaxel as second-line treatment in advanced non-small cell lung cancer: a study of the PMH phase II consortium.

Leighl NB, Laurie SA, Chen XE, Ellis P, Shepherd FA, Knox JJ, Goss G, Burkes RL, Pond GR, Dick C, Yen Y, Zwiebel JA, Moore MJ.

J Thorac Oncol. 2009 Sep;4(9):1163-9. doi: 10.1097/JTO.0b013e3181a949b2.


Phase I study of the c-raf-1 antisense oligonucleotide ISIS 5132 in combination with carboplatin and paclitaxel in patients with previously untreated, advanced non-small cell lung cancer.

Fidias P, Pennell NA, Boral AL, Shapiro GI, Skarin AT, Eder JP Jr, Kwoh TJ, Geary RS, Johnson BE, Lynch TJ, Supko JG.

J Thorac Oncol. 2009 Sep;4(9):1156-62. doi: 10.1097/JTO.0b013e3181b2793f.


Phase I/II trial of AEG35156 X-linked inhibitor of apoptosis protein antisense oligonucleotide combined with idarubicin and cytarabine in patients with relapsed or primary refractory acute myeloid leukemia.

Schimmer AD, Estey EH, Borthakur G, Carter BZ, Schiller GJ, Tallman MS, Altman JK, Karp JE, Kassis J, Hedley DW, Brandwein J, Xu W, Mak DH, LaCasse E, Jacob C, Morris SJ, Jolivet J, Andreeff M.

J Clin Oncol. 2009 Oct 1;27(28):4741-6. doi: 10.1200/JCO.2009.21.8172. Epub 2009 Aug 3.


Polyglutamine expansion in huntingtin alters its interaction with phospholipids.

Kegel KB, Sapp E, Alexander J, Valencia A, Reeves P, Li X, Masso N, Sobin L, Aronin N, DiFiglia M.

J Neurochem. 2009 Sep;110(5):1585-97. doi: 10.1111/j.1471-4159.2009.06255.x. Epub 2009 Jun 29.

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