Progress and challenges in RNA interference therapy for Huntington disease

Archives of Neurology

Volumn 66, Issue 8, 2009, Pages 933-938

  Harper, Scott Q ^a  

^a THE OHIO STATE UNIVERSITY WEXNER MEDICAL CENTER   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

HUNTINGTIN; MESSENGER RNA; MICRORNA; SHORT HAIRPIN RNA; SMALL INTERFERING RNA;

ALLELE; GAIT DISORDER; GENE SILENCING; HUMAN; HUNTINGTON CHOREA; MOTOR PERFORMANCE; NONHUMAN; NUCLEOTIDE SEQUENCE; PALLIATIVE THERAPY; PRIORITY JOURNAL; PROTEIN EXPRESSION; REVIEW; RNA INTERFERENCE; TRINUCLEOTIDE REPEAT;

ANIMALS; BASE PAIRING; DISEASE MODELS, ANIMAL; GENE SILENCING; GENE THERAPY; GENES, DOMINANT; GENETIC VECTORS; HUMANS; HUNTINGTON DISEASE; MICE; MICRORNAS; NERVE TISSUE PROTEINS; NUCLEAR PROTEINS; PROSPECTIVE STUDIES; RNA INTERFERENCE; RNA, MESSENGER; RNA-INDUCED SILENCING COMPLEX; TRANSFECTION;

EID: 68549104413     PISSN: 00039942     EISSN: 15383687     Source Type: Journal    
DOI: 10.1001/archneurol.2009.180     Document Type: Review

References (28)

1. Imarisio S, Carmichael J, Korolchuk V, et al. Huntington's disease: from pathology and genetics to potential therapies. Biochem J. 2008;412(2):191-209.
2. Phillips W, Shannon KM, Barker RA. The current clinical management of Huntington's disease. Mov Disord. 2008;23(11):1491-1504.
3. Shin JY, Fang ZH, Yu ZX, Wang CE, Li SH, Li XJ. Expression of mutant huntingtin in glial cells contributes to neuronal excitotoxicity. J Cell Biol. 2005;171 (6):1001-1012.
4. Zuccato C, Ciammola A, Rigamonti D, et al. Loss of huntingtin-mediated BDNF gene transcription in Huntington's disease. Science. 2001;293(5529):493-498.
5. Fire A, Xu S, Montgomery MK, Kostas SA, Driver SE, Mello CC. Potent and specific genetic interference by double-stranded RNA in Caenorhabditis elegans. Nature. 1998;391(6669):806-811.
6. Bartel DP. MicroRNAs: genomics, biogenesis, mechanism, and function. Cell. 2004;116(2):281-297.
7. Davidson BL, Harper SQ. Viral delivery of recombinant short hairpin RNAs. Methods Enzymol. 2005;392:145-173.
8. Zeng Y, Wagner EJ, Cullen BR. Both natural and designed micro RNAs can inhibit the expression of cognate mRNAs when expressed in human cells. Mol Cell. 2002;9(6):1327-1333.
9. Amarzguioui M, Lundberg P, Cantin E, Hagstrom J, Behlke MA, Rossi JJ. Rational design and in vitro and in vivo delivery of Dicer substrate siRNA. Nat Protoc. 2006;1(2):508-517.
10. Harper SQ, Staber PD, He X, et al. RNA interference improves motor and neu-ropathological abnormalities in a Huntington's disease mouse model. Proc Natl Acad Sci U S A. 2005;102(16):5820-5825.
11. Rodriguez-Lebron E, Denovan-Wright EM, Nash K, Lewin AS, Mandel RJ. Intrastriatal rAAV-mediated delivery of anti-huntingtin shRNAs induces partial reversal of disease progression in R6/1 Huntington's disease transgenic mice. Mol Ther. 2005;12(4):618-633.
12. Wang YL, Liu W, Wada E, Murata M, Wada K, Kanazawa I. Clinico-pathological rescue of a model mouse of Huntington's disease by siRNA. Neurosci Res. 2005; 53(3):241-249.
13. Machida Y, Okada T, Kurosawa M, Oyama F, Ozawa K, Nukina N. rAAV-mediated shRNA ameliorated neuropathology in Huntington disease model mouse. Biochem Biophys Res Commun. 2006;343(1):190-197.
14. DiFiglia M, Sena-Esteves M, Chase K, et al. Therapeutic silencing of mutant huntingtin with siRNA attenuates striatal and cortical neuropathology and behavioral deficits. Proc Natl Acad Sci U S A. 2007;104(43): 17204-17209.
15. Huang B, Schiefer J, Sass C, Landwehrmeyer GB, Kosinski CM, Kochanek S. High-capacity adenoviral vector-mediated reduction of huntingtin aggregate load in vitro and in vivo. Hum Gene Ther. 2007;18(4):303-311.
16. Franich NR, Fitzsimons HL, Fong DM, Klugmann M, During MJ, Young D. AAV vector-mediated RNAi of mutant huntingtin expression is neuroprotective in a novel genetic rat model of Huntington's disease. Mol Ther. 2008;16(5):947-956.
17. McBride JL, Boudreau RL, Harper SQ, et al. Artificial miRNAs mitigate shRNAmediated toxicity in the brain: implications for the therapeutic development of RNAi. Proc Natl Acad Sci U S A. 2008;105(15):5868-5873.
18. Drouet V, Perrin V, Hassig R, et al. Sustained effects of nonallele-specific Huntingtin silencing. Ann Neurol. 2009;65(3):276-285.
19. Boudreau RL, McBride JL, Martins I, et al. Nonallele-specific silencing of mutant and wild-type huntingtin demonstrates therapeutic efficacy in Huntington's disease mice. Mol Ther. 2009;17(6):1053-1063.
20. Denovan-Wright EM, Rodriguez-Lebron E, Lewin AS, Mandel RJ. Unexpected offtargeting effects of anti-huntingtin ribozymes and siRNA in vivo. Neurobiol Dis. 2008;29(3):446-455.
21. Marques JT, Williams BR. Activation of the mammalian immune system by siRNAs. Nat Biotechnol. 2005;23(11):1399-1405.
22. Dragatsis I, Levine MS, Zeitlin S. Inactivation of Hdh in the brain and testis results in progressive neurodegeneration and sterility in mice. Nat Genet. 2000; 26(3):300-306.
23. Zeitlin S, Liu JP, Chapman DL, Papaioannou VE, Efstratiadis A. Increased apoptosis and early embryonic lethality in mice nullizygous for the Huntington's disease gene homologue. Nat Genet. 1995;11(2):155-163.
24. van Bilsen PH, Jaspers L, Lombardi MS, Odekerken JC, Burright EN, Kaemmerer WF. Identification and allele-specific silencing of the mutant huntingtin allele in Huntington's disease patient-derived fibroblasts. Hum Gene Ther. 2008; 19(7):710-719.
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(9):e140. doi:10.1371/journal.pgen.0020140.
26. Ambrose CM, Duyao MP, Barnes G, et al. Structure and expression of the Huntington's disease gene: evidence against simple inactivation due to an expanded CAG repeat. Somat Cell Mol Genet. 1994;20(1):27-38.
27. Pfister EL, Kennington L, Straubhaar J, et al. Five siRNAs targeting three SNPs may provide therapy for three-quarters of Huntington's disease patients. Curr Biol. 2009;19(9):774-778. doi:10.1016/j.cub.2009.03.030.
28. Foust KD, Nurre E, Montgomery CL, Hernandez A, Chan CM, Kaspar BK. Intravascular AAV9 preferentially targets neonatal neurons and adult astrocytes. Nat Biotechnol. 2009;27(1):59-65.