Effect of Hypocretin Restoration on Wakefulness in Narcoleptic Patients

...

References

Adamantidis, A. R., Zhang, F., Aravanis, A. M., Deisseroth, K., & de Lecea, L. 2007. Neural substrates of awakening probed with optogenetic control of hypocretin neurons. Nature, 450, 420–424. doi: 10.1038/nature06310

Bartus, R. T., Weinberg, M. S., & Samulski, R. J. 2014. Parkinson’s disease gene therapy: Success by design meets failure by efficacy. Molecular Therapy, 22(3), 487-497. doi: 10.1038/mt.2013.281

Bourgin, P., Huitron-Resendiz, S., Spier, A. D., Fabre, V., Morte, B.,Criado, J. R., … & de Lecea, L. 2000. Hypocretin-1 modulates rapid eye movement sleep through activation of locus coeruleus neurons. Journal of Neuroscience, 20, 7760-7765.

Chemelli, R. M., Willie, J. T., Sinton, C. M., Elmquist, J. K., Scammell, T., Lee, C., … & Yanaqisawa, M. 1999. Narcolepsy in orexin knockout mice: molecular genetics of sleep regulation. Cell, 98(4), 437-451. doi: 10.1016/S0092-8674(00)81973-X

Coune, P. G., Schneider, B. L., & Aebischer, P. 2012. Parkinson’s disease: Gene therapies. Cold Spring Harbor Perspectives in Medicine, 2(4), 12-15. doi: 10.1101/cshperspect.a009431

Crocker, A., Espana, R. A., Papadopoulou, M., Saper, C. B., Faraco, J., Sakurai, T., ... & Scammell, T. E. 2005. Concomitant loss of dynorphin, NARP, and orexin in narcolepsy. Neurology, 65, 1184-1188. doi: 10.1212/01.wnl.0000168173.71940.ab

Espana, R. A., McCormack, S. L., Mochizuki, T., & Scammell, T. E. 2007. Running promotes wakefulness and increases cataplexy in orexin knockout mice. Sleep, 30(11), 1417–14125.

Griffey, M., Macauley, S. L., Ogilvie, J. M., Sands, M. S. 2005. AAV2-mediated ocular gene therapy for infantile neuronal ceroid lipofuscinosis. Molecular Therapy, 12, 413-421. doi: 10.1016/j.ymthe.2005.04.018

Hara, J., Beuckmann, C. T., Nambu, T., Willie, J. T., Chemelli, R. M., Sinton, C. M., … & Sakurai, T. 2001. Genetic ablation of orexin neurons in mice results in narcolepsy, hypophagia, and obesity. Neuron, 30(2), 345-354.

High, K. A., & Aubourg, P. 2011. rAAV human trial experience. Methods in Molecular Biology, 807, 429-457. doi: 10.1007/978-1-61779-370-7_18.

Kantor, S., Mochizuki, T., Lops, S. N., Ko, B., Clain, E., Clark, E., … & Scammell, T. E. 2013. Orexin Gene Therapy Restores the Timing and Maintenance of Wakefulness in Narcoleptic Mice. Sleep, 36(8), 1129-1138. doi: 10.5665/sleep.2870

LeWitt, P. A., Rezai, A. R., Leehey, M. A., Ojemann, S. G., Flaherty, A. W., Eskandar, E. N., … & Feigin, A. 2011. AAV2-GAD gene therapy for advanced Parkinson's disease: a double-blind, sham-surgery controlled, randomised trial. The Lancet. Neurology, 10(4), 309-319. doi: 10.1016/S1474-4422(11)70039-4

Lu H, Qu G, Yang X, Xu R, Xiao W. Systemic elimination of de novo capsid protein synthesis from replication-competent AAV contamination in the liver. 2011. Human Gene Therapy, 22(5), 625-632. doi: 10.1089/hum.2011.005

Mieda, M., Willie, J. T., Hara, J., Sinton, C. M., Sakurai, T., & Yanagisawa, M. 2004. Orexin peptides prevent cataplexy and improve wakefulness in an orexin neuron-ablated model of narcolepsy in mice. Proceedings of the National Academy of Sciences of the United States of America, 101(13), 4649-4654. doi: 10.1073/pnas.0400590101

Mitler, M. M., & Hayduk, R. 2002. Benefits and risks of pharmacotherapy for narcolepsy. Drug Safety, 25(11), 791-809. doi: 10.2165/00002018-200225110-00004

Mochizuki, T., Arrigoni, E., Marcus, J. N., Clark, E. L., Yamamoto, M., Honer, M., … & Scammell, T. E. 2011. Orexin receptor 2 expression in the posterior hypothalamus rescues sleepiness in narcoleptic mice. Proceedings of the National Acedmy of Sciences of the United States of America, 108(11), 4471–4476. doi: 10.1073/pnas.1012456108

Naumann, A., & Daum, I. 2003. Narcolepsy: Pathophysiology and neuropsychological changes. Behavioural Neurology, 14, 89-98. doi: 10.1155/2003/323060

Nishino, S. 2007. Clinical and neurobiological aspects of narcolepsy. Sleep Medicine, 8(4), 373-399. doi: 10.1016/j.sleep.2007.03.008

Peacock, J., & Benca, R. M. 2010. Narcolepsy: Clinical features, co-morbities and treatment. The Indian Journal of Medical Research, 131, 338-349.

Peyron, C., Tighe, D. K., van den Pol, A. N., de Lecea, L., Heller, H. C., Sutcliffe, J. G., & Kilduff, T. S. 1998. Neurons containing hypocretin (orexin) project to multiple neuronal systems. Journal of Neuroscience, 18, 9996 –10015.

Scammell, T. E. 2003. The neurobiology, diagnosis, and treatment of narcolepsy. Annuals of Neurology, 53(2), 154-166. doi: 10.1002/ana.10444

Thannickal, T. C., Moore, R. Y., Nienhuis, R., Ramanathan, L., Gulyani, S., Aldrich, M., … & Seigel, J. M. 2000. Reduced number of hypocretin neurons in human narcolepsy. Neuroscience, 27(3), 469-474. doi: 10.1016/S0896-6273(00)00058-1

Xiao, X. Li, J., & Samulski, J. R. 1998. Production of High-Titer Recombinant Adeno-Associated Virus Vectors in the Absence of Helper Adenovirus. Journal of Virology, 72(3), 2224-2232.