Six-month partial suppression of Huntingtin is well tolerated in the adult rhesus striatum

Brain

Volumn 135, Issue 4, 2012, Pages 1197-1209

  Grondin, Richard ^a   Kaytor, Michael D ^b   Ai, Yi ^a   Nelson, Peter T ^c   Thakker, Deepak R ^b   Heisel, Jennifer ^b   Weatherspoon, Marcy R ^b   Blum, Janelle L ^b   Burright, Eric N ^b   Zhang, Zhiming ^a   Kaemmerer, William F ^b  

^a UNIVERSITY OF KENTUCKY   (United States)

^b MEDTRONIC INC   (United States)

^c UNIVERSITY OF KENTUCKY   (United States)

Author keywords

adeno associated virus; Huntington's disease; non human primate; RNA interference; striatum

Indexed keywords

ADENOVIRUS VECTOR; HUNTINGTIN; MESSENGER RNA; SHORT HAIRPIN RNA;

ADVERSE OUTCOME; ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; ARTICLE; BRAIN TISSUE; CONTROLLED STUDY; CORPUS STRIATUM; FEMALE; GENE SILENCING; GENE TARGETING; HETEROZYGOSITY; HISTOPATHOLOGY; HUMAN; HUMAN CELL; HUNTINGTON CHOREA; IMMUNOHISTOCHEMISTRY; LOCOMOTION; LONG TERM POTENTIATION; MOTOR PERFORMANCE; MUTANT; NERVE DEGENERATION; NEUROPATHOLOGY; NONHUMAN; PRIORITY JOURNAL; PROTEIN EXPRESSION; PUTAMEN; RHESUS MONKEY; RNA INTERFERENCE; RNA VECTOR; SEROTYPE; WESTERN BLOTTING; WILD TYPE;

EID: 84860192454     PISSN: 00068950     EISSN: 14602156     Source Type: Journal    
DOI: 10.1093/brain/awr333     Document Type: Article

References (31)

1. Ai Y, Markesbery W, Zhang Z, Grondin R, Elseberry D, Gerhardt GA, et al. Intraputamenal infusion of GDNF in aged rhesus monkeys: distribution and dopaminergic effects. J Comp Neurol 2003; 461: 250-61. (Pubitemid 36583466)
2. Andersen AH, Zhang Z, Zhang M, Gash DM, Avison MJ. Age-associated changes in rhesus CNS composition identified by MRI. Brain Res 1999; 829: 90-8. (Pubitemid 29251687)
3. Auerbach W, Hurlbert MS, Hilditch-Maguire P, Wadghiri YZ, Wheeler VC, Cohen SI, et al. The HD mutation causes progressive lethal neurological disease in mice expressing reduced levels of huntingtin. Hum Mol Genet 2001; 10: 2515-23. (Pubitemid 33095002)
4. Aylward EH, Sparks BF, Field KM, Yallapragada V, Shpritz BD, Rosenblatt A, et al. Onset and rate of striatal atrophy in preclinical Huntington disease. Neurology 2004; 63: 66-72. (Pubitemid 38943780)
5. Boudreau RL, McBride JL, Martins I, Shen S, Xing Y, Carter BJ, et al. Nonallele-specific silencing of mutant and wild-type huntingtin demonstrates therapeutic efficacy in Huntington's disease mice. Mol Ther 2009; 17: 1053-63.
6. Colin E, Zala D, Liot G, Rangone H, Borrell-Pages M, Li XJ, et al. Huntingtin phosphorylation acts as a molecular switch for anterograde/retrograde transport in neurons. EMBO J 2008; 27: 2124-34.
7. 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: 300-6.
8. Drouet V, Perrin V, Hassig R, Dufour N, Auregan G, Alves S, et al. Sustained effects of nonallele-specific Huntingtin silencing. Ann Neurol 2009; 65: 276-85.
9. Duyao MP, Auerbach AB, Ryan A, Persichetti F, Barnes GT, McNeil SM, et al. Inactivation of the mouse Huntington's disease gene homolog Hdh. Science 1995; 269: 407-10.
10. Ferreira T, Rasband W. ImageJ User Guide IJ 1. 45m. 2011. http://rsb. info. nih. gov/ij/docs/guide/userguide-27. html#toc-Subsection-27. 13. (6 August 2011, date last accessed).
11. Gabery S, Murphy K, Schultz K, Loy CT, McCusker E, Kirik D, et al. Changes in key hypothalamic neuropeptide populations in Huntington disease revealed by neuropathological analyses. Acta Neuropathol 2010; 120: 777-88.
12. Godin JD, Colombo K, Molina-Calavita M, Keryer G, Zala D, Charrin BC, et al. Huntingtin is required for mitotic spindle orientation and mammalian neurogenesis. Neuron 2010; 67: 392-406.
13. Gore AC, Terasawa E. A role for norepinephrine in the control of puberty in the female rhesus monkey, Macaca mulatta. Endocrinology 1991; 129: 3009-17.
14. Grondin R, Cass WA, Zhang Z, Stanford JA, Gerhardt GA, Gash DM. GDNF increases stimulus-evoked dopamine release and motor speed in aged rhesus monkeys. J Neurosci 2003; 23: 1974-80. (Pubitemid 36314753)
15. Harper SQ, Staber PD, He X, Eliason SL, Martins IH, Mao Q, et al. RNA interference improves motor and neuropathological abnormalities in a Huntington's disease mouse model. Proc Natl Acad Sci USA 2005; 102: 5820-5. (Pubitemid 40559643)
16. Kaemmerer WF, Burright EN, Kaytor MD, Blum JL, Heisel J, Thakker DR, et al. A pilot study of the safety of suppressing wildtype huntingtin in the primate striatum. The 36th Annual Meeting of the Society for Neuroscience; 2006. Atlanta, GA, 2006.
17. Lombardi MS, Jaspers L, Spronkmans C, Gellera C, Taroni F, Di Maria E, et al. A majority of Huntington's disease patients may be treatable by individualized allele-specific RNA interference. Exp Neurol 2009; 217: 312-9.
18. McBride JL, Boudreau RL, Harper SQ, Staber PD, Monteys AM, Martins I, et al. Artificial miRNAs mitigate shRNA-mediated toxicity in the brain: implications for the therapeutic development of RNAi. Proc Natl Acad Sci USA 2008; 105: 5868-73. (Pubitemid 351758464)
19. McCarty DM, Fu H, Monahan PE, Toulson CE, Naik P, Samulski RJ. Adeno-associated virus terminal repeat (TR) mutant generates selfcomplementary vectors to overcome the rate-limiting step to transduction in vivo. Gene Ther 2003; 10: 2112-8. (Pubitemid 37536760)
20. Menalled LB, Miller S, Fitzpatrick J, Kudwa A, Morin C, Patry M, et al. Conditional knock-down of endogenous huntingtin in mice: Behavioral characterization. The 39th Annual Meeting of the Society for Neuroscience; 2009. Chicago, IL, 2009.
21. Sah DW, Aronin N. Oligonucleotide therapeutic approaches for Huntington disease. J Clin Invest 2011; 121: 500-7.
22. Sanftner LM, Sommer JM, Suzuki BM, Smith PH, Vijay S, Vargas JA, et al. AAV2-mediated gene delivery to monkey putamen: evaluation of an infusion device and delivery parameters. Exp Neurol 2005; 194: 476-83. (Pubitemid 40967005)
23. Sass M, Aronin N. RNA-and DNA-Based Therapies for Huntington's Disease. Neurobiology of Huntington's Disease: Applications to Drug Discovery. 2nd edn. Boca Raton, Florida: CRC Press; 2011.
24. Tabrizi SJ, Langbehn DR, Leavitt BR, Roos RA, Durr A, Craufurd D, et al. Biological and clinical manifestations of Huntington's disease in the longitudinal TRACK-HD study: cross-sectional analysis of baseline data. Lancet Neurol 2009; 8: 791-801.
25. Tenenbaum L, Chtarto A, Lehtonen E, Velu T, Brotchi J, Levivier M. Recombinant AAV-mediated gene delivery to the central nervous system. J Gene Med 2004; 6 (Suppl 1): S212-22. (Pubitemid 40228030)
26. Tigges J, Gordon TP, McClure HM, Hall EC, Peters A. Survival rate and life span of rhesus monkeys at the Yerkes regional primate research center. American Journal of Primatology 1988; 15: 263-73.
27. 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: 710-9.
28. Walton A, Scheib JL, McLean S, Zhang Z, Grondin R. Motor memory preservation in aged monkeys mirrors that of aged humans on a similar task. Neurobiol Aging 2008; 29: 1556-62.
29. Wang YL, Liu W, Wada E, Murata M, Wada K, Kanazawa I. Clinicopathological rescue of a model mouse of Huntington's disease by siRNA. Neurosci Res 2005; 53: 241-9. (Pubitemid 41463777)
30. Zuccato C, Cattaneo E. Role of brain-derived neurotrophic factor in Huntington's disease. Prog Neurobiol 2007; 81: 294-330. (Pubitemid 46528643)
31. Zuccato C, Valenza M, Cattaneo E. Molecular mechanisms and potential therapeutical targets in Huntington's disease. Physiol Rev 2010; 90: 905-81.