1) Saito Y, Yokota T, Mitani T, et al. Transgenic small interfering RNA halts amyotrophic lateral sclerosis in a mouse model. J Biol Chem. 2005; 280: 42826-30
|
|
|
2) Foust KD, Nurre E, Montgomery CL, et al. Intravascular AAV9 preferentially targets neonatal neurons and adult astrocytes. Nat Biotechnol. 2009; 27: 59-65
|
|
|
3) Brummelkamp TR, Bernards R, Agami R. A system for stable expression of short interfering RNAs in mammalian cells. Science. 2002; 296: 550-3
|
|
|
4) Lee NS, Dohjima T, Bauer G, et al. Expression of small interfering RNAs targeted against HIV-1 rev transcripts in human cells. Nat Biotechnol. 2002; 20: 500-5
|
|
|
5) Miyagishi M, Sumimoto H, Miyoshi H, et al. Optimization of an siRNA-expression system with an improved hairpin and its significant suppressive effects in mammalian cells. J Gene Med. 2004; 6: 715-23
|
|
|
6) Boudreau RL, Monteys AM, Davidson BL. Minimizing variables among hairpin-based RNAi vectors reveals the potency of shRNAs. RNA. 2008; 14: 1834-44
|
|
|
7) Xia H, Mao Q, Paulson HL, Davidson BL. siRNA-mediated gene silencing in vitro and in vivo. Nat Biotechnol. 2002; 20: 1006-10
|
|
|
8) Ely A, Naidoo T, Mufamadi S, et al. Expressed anti-HBV primary microRNA shuttles inhibit viral replication efficiently in vitro and in vivo. Mol Ther. 2008; 16: 1105-12
|
|
|
9) McBride JL, Boudreau RL, Harper SQ, et al. Artificial miRNAs mitigate shRNA-mediated toxicity in the brain: implications for the therapeutic development of RNAi. Proc Natl Acad Sci U S A. 2008; 105: 5868-73
|
|
|
10) Harper SQ, Staber PD, He X, et al. RNA interference improves motor and neuropathological abnormalities in a Huntingtonʼs disease mouse model. Proc Natl Acad Sci U S A. 2005; 102: 5820-5
|
|
|
11) Drouet V, Perrin V, Hassig R, et al. Sustained effects of nonallele-specific Huntingtin silencing. Ann Neurol. 2009; 65: 276-85
|
|
|
12) Franich NR, Fitzsimons HL, Fong DM, et al. AAV vector-mediated RNAi of mutant huntingtin expression is neuroprotective in a novel genetic rat model of Huntingtonʼs disease. Mol Ther. 2008; 16: 947-56
|
|
|
13) Rodriguez-Lebron E, Gouvion CM, Moore SA, et al. Allele-specific RNAi mitigates phenotypic progression in a transgenic model of Alzheimerʼs disease. Mol Ther. 2009; 17: 1563-73
|
|
|
14) Miller TM, Kaspar BK, Kops GJ, et al. Virus-delivered small RNA silencing sustains strength in amyotrophic lateral sclerosis. Ann Neurol. 2005; 57: 773-6
|
|
|
15) Ralph GS, Radcliffe PA, Day DM, et al. Silencing mutant SOD1 using RNAi protects against neurodegeneration and extends survival in an ALS model. Nat Med. 2005; 11: 429-33
|
|
|
16) Raoul C, Abbas-Terki T, Bensadoun JC, et al. Lentiviral-mediated silencing of SOD1 through RNA interference retards disease onset and progression in a mouse model of ALS. Nat Med. 2005; 11: 423-8
|
|
|
17) Singer O, Marr RA, Rockenstein E, et al. Targeting BACE1 with siRNAs ameliorates Alzheimer disease neuropathology in a transgenic model. Nat Neurosci. 2005; 8: 1343-9
|
|
|
18) Sapru MK, Yates JW, Hogan S, et al. Silencing of human alpha-synuclein in vitro and in rat brain using lentiviral-mediated RNAi. Exp Neurol. 2006; 198: 382-90
|
|
|
19) Sun HS, Jackson MF, Martin LJ, et al. Suppression of hippocampal TRPM7 protein prevents delayed neuronal death in brain ischemia. Nat Neurosci. 2009; 12: 1300-7
|
|
|
20) Pfeifer A, Eigenbrod S, Al-Khadra S, et al. Lentivector-mediated RNAi efficiently suppresses prion protein and prolongs survival of scrapie-infected mice. J Clin Invest. 2006; 116: 3204-10
|
|
|
21) White MD, Farmer M, Mirabile I, et al. Single treatment with RNAi against prion protein rescues early neuronal dysfunction and prolongs survival in mice with prion disease. Proc Natl Acad Sci U S A. 2008; 105: 10238-43
|
|
|
22) Jackson AL, Bartz SR, Schelter J, et al. Expression profiling reveals off-target gene regulation by RNAi. Nat Biotechnol. 2003; 21: 635-7
|
|
|
23) Birmingham A, Anderson EM, Reynolds A, et al. 3ʼ UTR seed matches, but not overall identity, are associated with RNAi off-targets. Nat Methods. 2006; 3: 199-204
|
|
|
24) Naito Y, Yoshimura J, Morishita S, et al. siDirect 2. 0: updated software for designing functional siRNA with reduced seed-dependent off-target effect. BMC Bioinformatics. 2009; 10: 392
|
|
|
25) Schwarz DS, Ding H, Kennington L, et al. Designing siRNA that distinguish between genes that differ by a single nucleotide. PLoS Genet. 2006; 2: e140
|
|
|
26) Alves S, Nascimento-Ferreira I, Auregan G, et al. Allele-specific RNA silencing of mutant ataxin-3 mediates neuroprotection in a rat model of Machado-Joseph disease. PLoS One. 2008; 3: e3341
|
|
|
27) Pfister EL, Kennington L, Straubhaar J, et al. Five siRNAs targeting three SNPs may provide therapy for three-quarters of Huntingtonʼs disease patients. Curr Biol. 2009; 19: 774-8
|
|
|
28) Li Y, Yokota T, Matsumura R, et al. Sequence-dependent and independent inhibition specific for mutant ataxin-3 by small interfering RNA. Ann Neurol. 2004; 56: 124-9
|
|
|
29) Kubodera T, Yamada H, Anzai M, et al. In Vivo Application of an RNAi Strategy for the Selective Suppression of a Mutant Allele. Hum Gene Ther. 2010 Jul 22. [Epub ahead of print]
|
|
|
30) Grimm D, Streetz KL, Jopling CL, et al. Fatality in mice due to oversaturation of cellular microRNA/short hairpin RNA pathways. Nature. 2006; 441: 537-41
|
|
|
31) Ehlert EM, Eggers R, Niclou SP, et al. Cellular toxicity following application of adeno-associated viral vector-mediated RNA interference in the nervous system. BMC Neurosci. 2010; 11: 20
|
|
|
32) Ulusoy A, Sahin G, Bjorklund T, et al. Dose optimization for long-term rAAV-mediated RNA interference in the nigrostriatal projection neurons. Mol Ther. 2009; 17: 1574-84
|
|
|
33) Giering JC, Grimm D, Storm TA, et al. Expression of shRNA from a tissue-specific pol II promoter is an effective and safe RNAi therapeutic. Mol Ther. 2008; 16: 1630-6
|
|
|
34) Chen Q, Butler D, Querbes W, et al. Lipophilic siRNAs mediate efficient gene silencing in oligodendrocytes with direct CNS delivery. J Control Release. 2010; 144: 227-32
|
|
|
35) Querbes W, Ge P, Zhang W, et al. Direct CNS delivery of siRNA mediates robust silencing in oligodendrocytes. Oligonucleotides. 2009; 19: 23-9
|
|
|
36) McCormack AL, Mak SK, Henderson JM, et al. α-synuclein suppression by targeted small interfering RNA in the primate substantia nigra. PLoS One. 2010; 5: e12122
|
|
|
37) Hino T, Yokota T, Ito S, et al. In vivo delivery of small interfering RNA targeting brain capillary endothelial cells. Biochem Biophys Res Commun. 2006; 340: 263-7
|
|
|
38) Campbell M, Kiang AS, Kenna PF, et al. RNAi-mediated reversible opening of the blood-brain barrier. J Gene Med. 2008; 10: 930-47
|
|
|
39) Fuest C, Bankstahl M, Winter P, et al. In vivo down-regulation of mouse brain capillary P-glycoprotein: a preliminary investigation. Neurosci Lett. 2009; 464: 47-51
|
|
|
40) Thakker DR, Natt F, Husken D, et al. Neurochemical and behavioral consequences of widespread gene knockdown in the adult mouse brain using nonviral RNA interference. Proc Natl Acad Sci U S A. 2004; 101: 17270-5
|
|
|
41) Guissouma H, Froidevaux MS, Hassani Z, et al. In vivo siRNA delivery to the mouse hypothalamus confirms distinct roles of TR beta isoforms in regulating TRH transcription. Neurosci Lett. 2006; 406: 240-3
|
|
|
42) Luo MC, Zhang DQ, Ma SW, et al. An efficient intrathecal delivery of small interfering RNA to the spinal cord and peripheral neurons. Mol Pain. 2005; 1: 1-8
|
|
|
43) Yoshitaka Uno, Wenying Piao, Kazutaka Nishina, et al. HDL Facilitates In Vivo Delivery of a-Tocopherol-Conjugated siRNA to the Brain. Hum Gene Ther. 2010; in press
|
|
|
44) Zhang Y, Zhang YF, Bryant J, et al. Intravenous RNA interference gene therapy targeting the human epidermal growth factor receptor prolongs survival in intracranial brain cancer. Clin Cancer Res. 2004; 10: 3667-77
|
|
|
45) Davidson TJ, Harel S, Arboleda VA, et al. Highly efficient small interfering RNA delivery to primary mammalian neurons induces MicroRNA-like effects before mRNA degradation. J Neurosci. 2004; 24: 10040-6
|
|
|
46) Kumar P, Wu H, McBride JL, et al. Transvascular delivery of small interfering RNA to the central nervous system. Nature. 2007; 448: 39-43
|
|
|