Erratum: Progress in gene therapy for neurological disorders (Nature Reviews Neurology (2013) 9 (277-291) DOI: 10.1038/nrneurol.2013.56);Progress in gene therapy for neurological disorders

Nature Reviews Neurology

Volumn 9, Issue 5, 2013, Pages 277-291

  Simonato, Michele ^a   Bennett, Jean ^b   Boulis, Nicholas M ^c   Castro, Maria G ^d   Fink, David J ^d   Goins, William F ^h   Gray, Steven J ^e   Lowenstein, Pedro R ^d   Vandenberghe, Luk H ^f   Wilson, Thomas J ^d   Wolfe, John H ^g   Glorioso, Joseph C ^h  

^a UNIVERSITY OF FERRARA   (Italy)

^b UNIVERSITY OF PENNSYLVANIA   (United States)

^c EMORY UNIVERSITY   (United States)

^d UNIVERSITY OF MICHIGAN   (United States)

^e UNIVERSITY OF NORTH CAROLINA   (United States)

^f HARVARD MEDICAL SCHOOL   (United States)

^g CHILDREN S HOSPITAL OF PHILADELPHIA   (United States)

^h UNIVERSITY OF PITTSBURGH   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

ANTISENSE OLIGONUCLEOTIDE; AROMATIC LEVO AMINO ACID DECARBOXYLASE; BETA INTERFERON; BRAIN DERIVED NEUROTROPHIC FACTOR; ENKEPHALIN; ERYTHROPOIETIN; GLIAL CELL LINE DERIVED NEUROTROPHIC FACTOR; GLUTAMATE DECARBOXYLASE; GUANOSINE TRIPHOSPHATE CYCLOHYDROLASE I; HUNTINGTIN; INTERLEUKIN 10; INTERLEUKIN 4; LENTIVIRUS VECTOR; NERVE GROWTH FACTOR; NEUROPEPTIDE Y; NEUROTROPHIN 3; NEURTURIN; PARVOVIRUS VECTOR; PLACEBO; POTASSIUM CHANNEL KV1.1; SMALL INTERFERING RNA; SOMATOMEDIN C; THYMIDINE KINASE; TYROSINE 3 MONOOXYGENASE; VIRUS VECTOR;

ABCD1 GENE; ADRENOLEUKODYSTROPHY; BRAIN FUNCTION; CENTRAL NERVOUS SYSTEM DISEASE; CLINICAL EFFECTIVENESS; DEGENERATIVE DISEASE; DISEASE COURSE; DOPAMINE METABOLISM; DRUG EFFICACY; DRUG MECHANISM; EPIGENETICS; EPILEPSY; GENE; GENE DELIVERY SYSTEM; GENE EXPRESSION REGULATION; GENE MUTATION; GENE SILENCING; GENE TARGETING; GENE THERAPY; GENE VECTOR; GENETIC DISORDER; GENETIC ENGINEERING; GENETIC PROCEDURES; GLIOBLASTOMA; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HERPES SIMPLEX VIRUS; HUMAN; INHERITANCE; LEBER CONGENITAL AMAUROSIS; LOSS OF FUNCTION MUTATION; LYSOSOME STORAGE DISEASE; NERVE CELL EXCITABILITY; NERVE DEGENERATION; NERVOUS SYSTEM DEVELOPMENT; NEUROLOGIC DISEASE; NEUROPATHIC PAIN; NEUROPATHY; NEUROPROTECTION; NONHUMAN; PAIN; PARKINSON DISEASE; PATHOPHYSIOLOGY; PERIPHERAL NERVOUS SYSTEM; PHASE 1 CLINICAL TRIAL (TOPIC); PHASE 2 CLINICAL TRIAL (TOPIC); POLYNEUROPATHY; PREVALENCE; PRIORITY JOURNAL; RETINA DEGENERATION; RETINA MACULA AGE RELATED DEGENERATION; REVIEW; SENSORY NERVE CELL; SENSORY NEUROPATHY; TRANSGENE; TUMOR SUPPRESSOR GENE; VIRUS CAPSID; VIRUS LOAD;

EID: 84877578223     PISSN: 17594758     EISSN: 17594766     Source Type: Journal    
DOI: 10.1038/nrneurol.2013.103     Document Type: Erratum

References (155)

1. Elsabahy, M., Nazarali, A. & Foldvari, M. Non-viral nucleic acid delivery: key challenges and future directions. Curr. Drug Deliv. 8, 235-244 (2011).
2. Goncalves, M. A. Adeno-associated virus: from defective virus to effective vector. Virol. J. 2, 43 (2005).
3. Matrai, J., Chuah, M. K. & VandenDriessche, T. Recent advances in lentiviral vector development and applications. Mol. Ther. 18, 477-490 (2010).
4. Nayak, S. & Herzog, R. W. Progress and prospects: immune responses to viral vectors. Gene Ther. 17, 295-304 (2010).
5. Foust, K. D. et al. Intravascular AAV9 preferentially targets neonatal neurons and adult astrocytes. Nat. Biotechnol. 27, 59-65 (2009).
6. Duque, S. et al. Intravenous administration of self-complementary AAV9 enables transgene delivery to adult motor neurons. Mol. Ther. 7, 1187-1196 (2009).
7. Gray, S. J. et al. Preclinical differences of intravascular AAV9 delivery to neurons and glia: a comparative study of adult mice and nonhuman primates. Mol. Ther. 19, 1058-1069 (2011).
8. Pulicherla, N. et al. Engineering liver-detargeted AAV9 vectors for cardiac and musculoskeletal gene transfer. Mol. Ther. 6, 1070-1078 (2011).
9. Bennett. et al. Further reduction in adenovirus vector-mediated liver transduction without largely affecting transgene expression in target organ by exploiting microrna-mediated regulation and the Cre-loxP recombination system. Mol. Pharm. 9, 3452-3463.
10. Chen, Y. H., Chang, M. & Davidson, B. L. Molecular signatures of disease brain endothelia provide new sites for CNS-directed enzyme therapy. Nat. Med. 15, 1215-1218 (2009).
11. Maheshri, N., Koerber, J. T., Kaspar, B. K. & Schaffer, D. V. Directed evolution of adeno-associated virus yields enhanced gene delivery vectors. Nat. Biotechnol. 24, 198-204 (2006). (Pubitemid 43221704)
12. Gray, S. J. et al. Directed evolution of a novel adeno-associated virus (AAV) vector that crosses the seizure-compromised blood-brain barrier (BBB). Mol. Ther. 18, 570-578 (2010).
13. Hermening, S., Kugler, S., Bahr, M. & Isenmann,S. Improved high-capacity adenoviral vectors for high-level neuron-restricted gene transfer to the CNS. J. Virol. Methods 136, 30-37 (2006). (Pubitemid 44051573)
14. Gray, S. J. et al. Optimizing promoters for recombinant adeno-associated virus-mediated gene expression in the peripheral and central nervous system using self-complementary vectors. Hum. Gene Ther. 9, 1143-1153 (2011).
15. Qin, J. Y. et al. Systematic comparison of constitutive promoters and the doxycycline-inducible promoter. PLoS ONE 5, e10611 (2010).
16. Husain, T., Passini, M. A., Parente, M. K., Fraser, N. W. & Wolfe, J. H. Long-term AAV vector gene and protein expression in mouse brain from a small pan-cellular promoter is similar to neural cell promoters. Gene Ther. 16, 927-932 (2009).
17. Kugler, S. et al. Neuron-specific expression of therapeutic proteins: evaluation of different cellular promoters in recombinant adenoviral vectors. Mol. Cell Neurosci. 17, 78-96 (2001). (Pubitemid 32113540)
18. Peel, A. L., Zolotukhin, S., Schrimsher, G. W., Muzyczka, N. & Reier, P. J. Efficient transduction of green fluorescent protein in spinal cord neurons using adeno-associated virus vectors containing cell type-specific promoters. Gene Ther. 4, 16-24 (1997). (Pubitemid 27073218)
19. Kügler, S., Lingor, P., Schöll, U., Zolotukhin, S. & Bähr, M. Differential transgene expression in brain cells in vivo and in vitro from AAV-2 vectors with small transcriptional control units. Virology 311, 89-95 (2003). (Pubitemid 36794213)
20. Lee, Y., Messing, A., Su, M. & Brenner, M. GFAP promoter elements required for region-specific and astrocyte-specific expression. Glia 56, 481-493 (2008).
21. den Hollander, A. I., Black, A., Bennett, J. & Cremers, F. P. Lighting a candle in the dark: advances in genetics and gene therapy of recessive retinal dystrophies. J. Clin. Invest. 120, 3042-3053 (2010).
22. Farrar, G. J., Millington-Ward, S., Chadderton, N., Humphries, P. & Kenna, P. F. Gene-based therapies for dominantly inherited retinopathies. Gene Ther. 19, 137-144 (2012).
23. Millington-Ward, S. et al. Suppression and replacement gene therapy for autosomal dominant disease in a murine model of dominant retinitis pigmentosa. Mol. Ther. 19, 642-649 (2011).
24. Jiang, L. et al. Long-term RNA interference gene therapy in a dominant retinitis pigmentosa mouse model. Proc. Natl Acad. Sci. USA 108, 18476-18481 (2011).
25. Lai, Y. K. et al. Potential long-term inhibition of ocular neovascularisation by recombinant adeno-associated virus-mediated secretion gene therapy. Gene Ther. 9, 804-813 (2002). (Pubitemid 34679308)
26. Jomary, C. & Jones, S. E. Induction of functional photoreceptor phenotype by exogenous Crx expression in mouse retinal stem cells. Invest. Ophthalmol. Vis. Sci. 49, 429-437 (2008).
27. Gubbels, S. P., Woessner, D. W., Mitchell, J. C., Ricci, A. J. & Brigande, J. V. Functional auditory hair cells produced in the mammalian cochlea by in utero gene transfer. Nature 455, 537-541 (2008).
28. Izumikawa, M. et al. Auditory hair cell replacement and hearing improvement by Atoh1 gene therapy in deaf mammals. Nat. Med. 11, 271-276 (2005). (Pubitemid 40460554)
29. Busskamp, V., Picaud, S., Sahel, J. A. & Roska, B. Optogenetic therapy for retinitis pigmentosa. Gene Ther. 19, 169-175 (2012).
30. Ashtari, M. et al. The human visual cortex responds to gene therapy-mediated recovery of retinal function. J. Clin. Invest. 121, 2160-2168 (2011).
31. Hauswirth, W. W. et al. Treatment of leber congenital amaurosis due to RPE65 mutations by ocular subretinal injection of adeno-associated virus gene vector: short-term results of a phase I trial. Human Gene Ther. 19, 979-990 (2008).
32. Bainbridge, J. W. et al. Effect of gene therapy on visual function in Leber's congenital amaurosis. N. Engl. J. Med. 358, 2231-2239 (2008). (Pubitemid 351724452)
33. Maguire, A. M. et al. Safety and efficacy of gene transfer for Leber's congenital amaurosis. N. Engl. J. Med. 358, 2240-2248 (2008). (Pubitemid 351724453)
34. Cremers, F. P., van de Pol., D. J., van Kerkhoff, L. P., Wieringa, B. & Ropers, H. H. Cloning of a gene that is rearranged in patients with choroideraemia. Nature 347, 674-677 (1990).
35. Dryja, T. P. et al. A point mutation of the rhodopsin gene in one form of retinitis pigmentosa. Nature 343, 364-366 (1990).
36. Farrar, G. J. et al. Autosomal dominant retinitis pigmentosa: linkage to rhodopsin and evidence for genetic heterogeneity. Genomics 8, 35-40 (1990). (Pubitemid 20302274)
37. Akil, O. et al. Restoration of hearing in the VGLUT3 knockout mouse using virally mediated gene therapy. Neuron 75, 283-293 (2012).
38. Lentz, J. J. et al. Rescue of hearing and vestibular function by antisense oligonucleotides in a mouse model of human deafness. Nat. Med. http://dx/doi.org/10.1038/nm.3106.
39. McIntyre, J. C. et al. Gene therapy rescues cilia defects and restores olfactory function in a mammalian ciliopathy model. Nat. Med. 18, 1423-1428 (2012).
40. Glorioso, J. C. & Fink, D. J. Herpes vector-mediated gene transfer in treatment of diseases of the nervous system. Annu. Rev. Microbiol. 58, 253-271 (2004). (Pubitemid 39551987)
41. Mata, M., Glorioso, J. C. & Fink, D. J. Gene therapy: novel treatments for polyneuropathy and chronic pain. Curr. Neurol. Neurosci. Rep. 4, 1-2 (2004). (Pubitemid 38899045)
42. Goss, J. R. et al. HSV delivery of a ligand-regulated endogenous ion channel gene to sensory neurons results in pain control following channel activation. Mol. Ther. 19, 500-506 (2011).
43. Goss, J. R. et al. Antinociceptive effect of a genomic herpes simplex virus-based vector expressing human proenkephalin in rat dorsal root ganglion. Gene Ther. 8, 551-556 (2001). (Pubitemid 32409530)
44. Goss, J. R. et al. Herpes vector-mediated expression of proenkephalin reduces bone cancer pain. Ann. Neurol. 52, 662-665 (2002). (Pubitemid 35246874)
45. Hao, S., Mata, M., Goins, W., Glorioso, J. C. & Fink, D. J. Transgene-mediated enkephalin release enhances the effect of morphine and evades tolerance to produce a sustained antiallodynic effect in neuropathic pain. Pain 102, 135-142 (2003). (Pubitemid 36298869)
46. Hao, S. et al. Gene transfer of glutamic acid decarboxylase reduces neuropathic pain. Ann. Neurol. 57, 914-918 (2005). (Pubitemid 40798630)
47. Chattopadhyay, M., Mata, M. & Fink, D. J. Vector-mediated release of GABA attenuates pain-related behaviors and reduces NaV1.7 in DRG neurons. Eur. J. Pain 15, 913-920 (2011).
48. Hao, S., Mata, M., Glorioso, J. C. & Fink, D. J. HSV-mediated expression of interleukin-4 in dorsal root ganglion neurons reduces neuropathic pain. Mol. Pain 2, 6 (2006).
49. Zhou, Z., Peng, X., Hao, S., Fink, D. J. & Mata, M. HSV-mediated transfer of interleukin-10 reduces inflammatory pain through modulation of membrane tumor necrosis factor α in spinal cord microglia. Gene Ther. 15, 183-190 (2008). (Pubitemid 351150041)
50. Lau, D. et al. Herpes simplex virus vector-mediated expression of interleukin-10 reduces below-level central neuropathic pain after spinal cord injury. Neurorehabil. Neural Repair 26, 889-897 (2012).
51. Goss, J. R. et al. Herpes simplex-mediated gene transfer of nerve growth factor protects against peripheral neuropathy in streptozotocin-induced diabetes in the mouse. Diabetes 51, 2227-2232 (2002). (Pubitemid 34729029)
52. Chattopadhyay, M. et al. Protective effect of HSV-mediated gene transfer of nerve growth factor in pyridoxine neuropathy demonstrates functional activity of trkA receptors in large sensory neurons of adult animals. Eur. J. Neurosci. 17, 732-740 (2003). (Pubitemid 36315243)
53. Chattopadhyay, M. et al. Protective effect of herpes simplex virus-mediated neurotrophin gene transfer in cisplatin neuropathy. Brain 127, 929-939 (2004). (Pubitemid 38478536)
54. Chattopadhyay, M., Walter, C., Mata, M. & Fink, D. J. Neuroprotective effect of herpes simplex virus-mediated gene transfer of erythropoietin in hyperglycemic dorsal root ganglion neurons. Brain 132, 879-888 (2009).
55. Chattopadhyay, M. et al. Prolonged preservation of nerve function in diabetic neuropathy in mice by herpes simplex virus-mediated gene transfer. Diabetologia 50, 1550-1558 (2007). (Pubitemid 46879025)
56. Wu, Z., Mata, M. & Fink, D. J. Prolonged regulatable expression of EPO from an HSV vector using the LAP2 promoter element. Gene Ther. 19, 1107-1113 (2012).
57. Fink, D. J. et al. Gene therapy for pain: results of a phase I clinical trial. Ann. Neurol. 70, 207-212 (2011).
58. Wolfe, D., Mata, M. & Fink, D. J. A human trial of HSV-mediated gene transfer for the treatment of chronic pain. Gene Therapy 14, 455-460 (2009).
59. Harper, S. Q. et al. RNA interference improves motor and neuropathological abnormalities in a Huntington's disease mouse model. Proc. Natl Acad. Sci. USA 16, 5820-5825 (2005). (Pubitemid 40559643)
60. Wolfe, J. H. Gene therapy in large animal models of human genetic diseases. ILAR J. 50, 107-111 (2009).
61. Vite, C. H. et al. Effective gene therapy for an inherited CNS disease in a large animal model. Ann. Neurol. 57, 355-364 (2005). (Pubitemid 40343957)
62. Berges, B. K. et al. Widespread correction of lysosomal storage in the mucopolysaccharidosis type VII mouse brain with a herpes simplex virus type 1 vector expressing β-glucuronidase. Mol. Ther. 13, 859-869 (2006).
63. Cearley, C. N. & Wolfe, J. H. A single injection of an adeno-associated virus vector into nuclei with divergent connections results in widespread vector distribution in the brain and global correction of a neurogenetic disease. J. Neurosci. 27, 9928-9940 (2007). (Pubitemid 47404014)
64. Baek, R. C. et al. AAV-mediated gene delivery in adult GM1-gangliosidosis mice corrects lysosomal storage in CNS and improves survival. PloS ONE 5, e13468 (2010).
65. Lui, G., Martins, I. H., Wemmie, J. A., Chiorini, J. A. & Davidson, B. L. Functional correction of CNS phenotypes in a lysosomal storage disease model using adeno-associated virus type 4 vectors. J. Neurosci. 25, 9321-9327 (2005). (Pubitemid 41464744)
66. Donsante, A. et al. ATP7A gene addition to the choroid plexus results in long-term rescue of the lethal copper transport defect in a Menkes disease mouse model. Mol. Ther. 19, 2114-2123 (2011).
67. Watson, D. J. & Wolfe, J. H. Lentiviral vectors for gene transfer to the central nervous system. Applications in lysosomal storage disease animal models. Meth. Mol. Med. 76, 383-403 (2003).
68. Cearley, C. N. & Wolfe, J. H in Encyclopedia of Neuroscience (ed. Squire, L. R.) 179-188 (Academic Press, Oxford, 2009).
69. Ellinwood, N. M. et al. Safe, efficient, and reproducible gene therapy of the brain in the dog models of Sanfilippo and Hurler syndromes. Mol. Ther. 19, 251-259 (2011).
70. Leone, P. et al. Aspartoacylase gene transfer to the mammalian central nervous system with therapeutic implications for Canavan disease. Ann. Neurol. 48, 27-38 (2000). (Pubitemid 30432428)
71. Worgall, S. et al. Treatment of late infantile neuronal ceroid lipofuscinosis by CNS administration of a serotype 2 adeno-associated virus expressing CLN2 cDNA. Human Gene Ther. 19, 463-474 (2008). (Pubitemid 351748791)
72. Cartier, N. et al. Hematopoietic stem cell gene therapy with a lentiviral vector in X-linked adrenoleukodystrophy. Science 326, 818-823 (2009).
73. Brookmeyer R. & Gray, S. Method for projecting the incidence and prevalence of chronic diseases in aging populations; application to Alzheimer's disease. Stat. Med. 19, 1481-1493 (2000). (Pubitemid 30346241)
74. Holtzman, D. M. et al. Alzheimer's disease: the challenge of the second century. Sci. Transl. Med. 3, 77sr71 (2011).
75. Wirdefeldt, K., Adami H.-O., Cole, P., Trichopoulos, D. & Mandel, J. Epidemiology and etiology of Parkinson's disease; a review of the evidence. Eur. J. Epidemiol. 26, S1-S58 (2011).
76. Siddique, T. & Ajroud-Driss, S. Familial amyotrophic lateral sclerosis, a historical perspective. Acta Myol. 2, 117-120 (2011).
77. [No authors listed] A novel gene containing a trinucleotide repeat that is expanded and unstable on Huntington's disease chromosomes. The Huntington's Disease Collaborative Research Group. Cell 72, 971-983 (1993).
78. Lang, A. E. & Lozano, A. M. Parkinson's disease. Second of two parts. N. Engl. J. Med. 339, 1130-1143 (1998). (Pubitemid 28465762)
79. Azzouz, M. et al. Multicistronic lentiviral vector-mediated striatal gene transfer of aromatic l-amino acid decarboxylase, tyrosine hydroxylase, and GTP cyclohydrolase I induces sustained transgene expression, dopamine production, and functional improvement in a rat model of Parkinson's disease. J. Neurosci. 22, 10302-10312 (2002). (Pubitemid 35416868)
80. Jarraya, B. et al. Dopamine gene therapy for Parkinson's disease in a nonhuman primate without associated dyskinesia. Sci. Transl. Med. 1, 2ra4 (2009).
81. Leff, S. E., Spratt, S. K., Snyder, R. O. & Mandel, R. J. Long-term restoration of striatal l-aromatic amino acid decarboxylase activity using recombinant adeno-associated viral vector gene transfer in a rodent model of Parkinson's disease. Neuroscience 92, 185-196 (1999). (Pubitemid 29249029)
82. Bankiewicz, K. S. et al. Long-term clinical improvement in MPTP-lesioned primates after gene therapy with AAV-hAADC. Mol. Ther. 14, 564-570 (2006). (Pubitemid 44296090)
83. Christine, C. W. et al. Safety and tolerability of putaminal AADC gene therapy for Parkinson disease. Neurology 73, 1662-1669 (2009).
84. Muramatsu, S. et al. A phase I study of aromatic L-amino acid decarboxylase gene therapy for Parkinson's disease. Mol. Ther. 18, 1731-1735 (2010).
85. Levy, R. et al. Lidocaine and muscimol microinjections in subthalamic nucleus reverse Parkinsonian symptoms. Brain 124, 2105-2118 (2001). (Pubitemid 32945907)
86. Luo, J. et al. Subthalamic GAD gene therapy in a Parkinson's disease rat model. Science 298, 425-429 (2002). (Pubitemid 35176353)
87. Emborg, M. E. et al. Subthalamic glutamic acid decarboxylase gene therapy: changes in motor function and cortical metabolism. J. Cereb. Blood Flow Metab. 27, 501-509 (2007).
88. LeWitt, P. A. et al. AAV2-GAD gene therapy for advanced Parkinson's disease: a double-blind, sham-surgery controlled, randomised trial. Lancet Neurol. 10, 309-319 (2011).
89. Oiwa, Y., Yoshimura, R., Nakai, K. & Itakura, T. Dopaminergic neuroprotection and regeneration by neurturin assessed by using behavioral, biochemical and histochemical measurements in a model of progressive Parkinson's disease. Brain Res. 947, 271-283 (2002).
90. Kordower, J. H. et al. Delivery of neurturin by AAV2 (CERE-120)-mediated gene transfer provides structural and functional neuroprotection and neurorestoration in MPTP-treated monkeys. Ann. Neurol. 60, 706-715 (2006). (Pubitemid 46048488)
91. Marks, W. J., Jr. et al. Safety and tolerability of intraputaminal delivery of CERE-120 (adeno-associated virus serotype 2-neurturin) to patients with idiopathic Parkinson's disease: an open-label, phase I trial. Lancet Neurol. 7, 400-408 (2008).
92. Marks, W. J. Jr. et al. Gene delivery of AAV2-neurturin for Parkinson's disease: a double-blind, randomised, controlled trial. Lancet Neurol. 9, 1164-1172 (2010).
93. Fisher, R. S. et al. Epileptic seizures and epilepsy: definitions proposed by the International League Against Epilepsy (ILAE) and the International Bureau for Epilepsy (IBE). Epilepsia 46, 470-472 (2005). (Pubitemid 40470667)
94. Berg, A. T. et al. Revised terminology and concepts for organization of seizures and epilepsies: report of the ILAE commission on classification and terminology, 2005-2009. Epilepsia 51, 676-685 (2010).
95. Galanopoulou, A. S. et al. Identification of new epilepsy treatments: issues in preclinical methodology. Epilepsia 53, 571-582 (2012).
96. Simonato, M., Tongiorgi, E. & Kokaia, M. Angels and demons: neurotrophic factors and epilepsy. Trends Pharmacol. Sci. 27, 631-638 (2006). (Pubitemid 44738088)
97. Paradiso, B. et al. Localized delivery of fibroblast growth factor-2 and brain-derived neurotrophic factor reduces spontaneous seizures in an epilepsy model. Proc. Natl Acad. Sci. USA 106, 7191-7196 (2009).
98. Bovolenta, R. et al. Hippocampal FGF-2 and BDNF overexpression attenuates epileptogenesis-associated neuroinflammation and reduces spontaneous recurrent seizures. J. Neuroinflammation 7, 81 (2010).
99. Paradiso, B. et al. Localized overexpression of FGF-2 and BDNF in hippocampus reduces mossy fiber sprouting and spontaneous seizures up to 4 weeks after pilocarpine-induced status epilepticus. Epilepsia 52, 572-578 (2011).
100. Haberman, R. et al. Therapeutic liabilities of in vivo viral vector tropism: adeno-associated virus vectors, NMDAR1 antisense, and focal seizure sensitivity. Mol. Ther. 6, 495-500 (2002). (Pubitemid 35214871)
101. Raol, Y. H. et al. Enhancing GABAA receptor α1 subunit levels in hippocampal dentate gyrus inhibits epilepsy development in an animal model of temporal lobe epilepsy. J. Neurosci. 26, 11342-11346 (2006). (Pubitemid 44772163)
102. Wykes, R. C. et al. Optogenetic and potassium channel gene therapy in a rodent model of focal neocortical epilepsy. Sci. Transl. Med. 161, 161ra152 (2012).
103. Haberman, R. P., Samulski, R. J. & McCown, T. J. Attenuation of seizures and neuronal death by adeno-associated virus vector galanin expression and secretion. Nat. Med. 9, 1076-1080 (2003). (Pubitemid 37021789)
104. Lin, E. J. et al. Recombinant AAV-mediated expression of galanin in rat hippocampus suppresses seizure development. Eur. J. Neurosci. 18, 2087-2092 (2003).
105. Richichi, C. et al. Anticonvulsant and antiepileptogenic effects mediated by adeno-associated virus vector neuropeptide Y expression in the rat hippocampus. J. Neurosci. 24, 3051-3059 (2004). (Pubitemid 38405752)
106. McCown, T. J. Adeno-associated virus-mediated expression and constitutive secretion of galanin suppresses limbic seizure activity in vivo. Mol. Ther. 14, 63-68 (2006). (Pubitemid 43899490)
107. Foti, S., Haberman, R. P., Samulski, R. J. & McCown, T. J. Adeno-associated virus-mediated expression and constitutive secretion of NPY or NPY13-36 suppresses seizure activity in vivo. Gene Ther. 14, 1534-1536 (2007). (Pubitemid 47570712)
108. Noe, F. et al. Neuropeptide Y gene therapy decreases chronic spontaneous seizures in a rat model of temporal lobe epilepsy. Brain 131, 1506-1515 (2008). (Pubitemid 351806462)
109. Sorensen, A. T. et al. Hippocampal NPY gene transfer attenuates seizures without affecting epilepsy-induced impairment of LTP. Exp. Neurol. 215, 328-333 (2009).
110. Noe, F. et al. Anticonvulsant effects and behavioural outcomes of rAAV serotype 1 vector-mediated neuropeptide Y overexpression in rat hippocampus. Gene Ther. 17, 643-652 (2010).
111. Woldbye, D. P. et al. Adeno-associated viral vector-induced overexpression of neuropeptide Y Y2 receptors in the hippocampus suppresses seizures. Brain 133, 2778-2788 (2010).
112. Grossman, S. A. et al. Survival of patients with newly diagnosed glioblastoma treated with radiation and temozolomide in research studies in the United States. Clin. Cancer Res. 8, 2443-2449 (2010).
113. Castro, M. et al. Gene therapy and targeted toxins for glioma. Curr. Gene Ther. 11, 155-180 (2012).
114. Ram, Z. et al. Therapy of malignant brain tumors by intratumoral implantation of retroviral vector-producing cells. Nat. Med. 3, 1354-1361 (1997). (Pubitemid 28011035)
115. Izquierdo, M. et al. Human malignant brain tumor response to herpes simplex thymidine kinase (HSVtk)/ganciclovir gene therapy. Gene Ther. 3, 491-495 (1996). (Pubitemid 26197538)
116. Shand, N. et al. A phase 1-2 clinical trial of gene therapy for recurrent glioblastoma multiforme by tumor transduction with the herpes simplex thymidine kinase gene followed by ganciclovir. GLI328 European-Canadian Study Group. Hum. Gene Ther. 10, 2325-2335 (1999). (Pubitemid 29459843)
117. Klatzmann, D. et al. A phase I/II study of herpes simplex virus type 1 thymidine kinase "suicide" gene therapy for recurrent glioblastoma. Study Group on Gene Therapy for Glioblastoma. Hum. Gene Ther. 9, 2595-2604 (1998). (Pubitemid 29004632)
118. Prados, M. D. et al. Treatment of progressive or recurrent glioblastoma multiforme in adults with herpes simplex virus thymidine kinase gene vector-producer cells followed by intravenous ganciclovir administration: a phase I/II multi-institutional trial. J. Neurooncol 65, 269-278 (2003). (Pubitemid 37526819)
119. Rainov, N. G. A phase III clinical evaluation of herpes simplex virus type 1 thymidine kinase and ganciclovir gene therapy as an adjuvant to surgical resection and radiation in adults with previously untreated glioblastoma multiforme. Hum. Gene Ther. 11, 2389-2401 (2000).
120. Trask, T. W. et al. Phase I study of adenoviral delivery of the HSV-tk gene and ganciclovir administration in patients with current malignant brain tumors. Mol. Ther. 1, 195-203 (2000).
121. Germano, I. M., Fable, J., Gultekin, S. H. & Silvers, A. Adenovirus/herpes simplex-thymidine kinase/ganciclovir complex: preliminary results of a phase I trial in patients with recurrent malignant gliomas. J. Neurooncol 65, 279-289 (2003). (Pubitemid 37526820)
122. Immonen, A. et al. AdvHSV-tk gene therapy with intravenous ganciclovir improves survival in human malignant glioma: a randomised, controlled study. Mol. Ther. 10, 967-972 (2004). (Pubitemid 39419922)
123. Sandmair, A. M. et al. Thymidine kinase gene therapy for human malignant glioma, using replication-deficient retroviruses or adenoviruses. Hum. Gene Ther. 11, 2197-2205 (2000).
124. Good Clinical Practice Inspectors Working Group. Reflection paper on GCP compliance in relation to trial master files (paper and/or electronic) of management, audit and inspection of clinical trials. European Medicines Agency [online], http://www.ema.europa.eu/docs/en-GB/document-library/Scientific- guideline/2013/02/WC500138893.pdf (2013).
125. Chiocca, E. A. et al. A phase I trial of Ad.hIFN-β gene therapy for glioma. Mol. Ther. 16, 618-626 (2008). (Pubitemid 351315003)
126. Lang, F. F. et al. Phase I trial of adenovirus-mediated p53 gene therapy for recurrent glioma: biological and clinical results. J. Clin. Oncol. 21, 2508-2518 (2003). (Pubitemid 46606332)
127. Papanastassiou, V. et al. The potential for efficacy of the modified (ICP 34.5-herpes simplex virus HSV1716 following intratumoural injection into human malignant glioma: a proof of principle study. Gene Ther. 9, 398-406 (2002). (Pubitemid 34414038)
128. Chiocca, E. A. et al. A phase I open-label, dose-escalation, multi-institutional trial of injection with an E1B-attenuated adenovirus, ONYX-015, into the peritumoral region of recurrent malignant gliomas, in the adjuvant setting. Mol. Ther. 10, 958-966 (2004). (Pubitemid 39419921)
129. Forsyth, P. et al. A phase I trial of intratumoral administration of reovirus in patients with histologically confirmed recurrent malignant gliomas. Mol. Ther. 16, 627-632 (2008). (Pubitemid 351315004)
130. Markert, J. M. et al. Phase Ib trial of mutant herpes simplex virus G207 inoculated pre-and post-tumor resection for recurrent GBM. Mol. Ther. 17, 199-207 (2009).
131. Yun, J. et al. A novel adenoviral vector labeled with superparamagnetic iron oxide nanoparticles for real-time tracking of viral delivery. J. Clin. Neurosci. 19, 875-880 (2012).
132. Vande Velde, G. et al. Evaluation of the specificity and sensitivity of ferritin as an MRI reporter gene in the mouse brain using lentiviral and adeno-associated viral vectors. Gene Ther. 18, 594-605 (2011).
133. Thevenot, E. et al. Targeted delivery of self-complementary adeno-associated virus serotype 9 to the brain, using magnetic resonance imaging-guided focused ultrasound. Hum. Gene Ther. 23, 1144-1155 (2012).
134. Markert, J. M. et al. Conditionally replicating herpes simplex virus mutant, G207 for the treatment of malignant glioma: results of a phase I trial. Gene Ther. 7, 867-874 (2000). (Pubitemid 30303584)
135. Rampling, R. et al. Toxicity evaluation of replication-competent herpes simplex virus (ICP 34.5 null mutant 1716) in patients with recurrent malignant glioma. Gene Ther. 7, 859-866 (2000). (Pubitemid 30303583)
136. Freeman, A. I. et al. Phase I/II trial of intravenous NDV-HUJ oncolytic virus in recurrent glioblastoma multiforme. Mol. Ther. 13, 221-228 (2006). (Pubitemid 41763501)
137. Ali, S. et al. Combined immunostimulation and conditional cytotoxic gene therapy provide long-term survival in a large glioma model. Cancer Res. 65, 7194-7204 (2005). (Pubitemid 41161250)
138. Chiocca, E. A. et al. Phase IB study of gene-mediated cytotoxic immunotherapy adjuvant to up-front surgery and intensive timing radiation for malignant glioma. J. Clin. Oncol. 29, 3611-3619 (2011).
139. Palu, G. et al. Gene therapy of glioblastoma multiforme via combined expression of suicide and cytokine genes: a pilot study in humans. Gene Ther. 6, 330-337 (1999). (Pubitemid 29132336)
140. Curtin, J. F. et al. HMGB1 mediates endogenous TLR2 activation and brain tumor regression. PLoS Med. 6, e10 (2009).
141. US National Institutes of Health. ClinicalTrials.gov [online], http://clinicaltrials.gov/ct2/show/NCT01811992 (2013).
142. Kells, A. P. et al. Regeneration of the MPTP-lesioned dopaminergic system after convection-enhanced delivery of AAV2-GDNF. J. Neurosci. 28, 9567-9577 (2010).
143. Bartus, R. T. et al. The safety and feasibility of combined substantia nigral putaminal stereotactic targetingof AAV2-neurturin (CERE120) in Parkinson's disease. Neurology (in press).
144. McBride, J. L. et al. Preclinical safety of RNAi-mediated HTT suppression in the rhesus macaque as a potential therapy for Huntington's disease. Mol. Ther. 12, 2152-2162 (2011).
145. Ramaswamy, S. et al. Intrastriatal CERE120 (AAV-neurturin) protects striatal and cortical neurons and delays motor deficits in a transgenic mouse model of Huntington's disease. Neurobiol. Dis. 34, 40-50 (2009).
146. Arvanitakis, Z. et al. Interim data from a phase 1 clinical trial of AAV-NGF (CERE-110) gene delivery in Alzheimer's disease [abstract P05.071]. Neurology 68 (Suppl. 1), A233-A234 (2007).
147. Nagahara, A. H. et al. Neuroprotective effects of brain-derived neurotrophic factor in rodent andprimate models of Alzheimer's disease. Nat. Med. 3, 331-337 (2009).
148. Suzuki, M. et al. GDNF secreting human neural progenitor cells protect dying motor neurons, but not their projection to muscle, in a rat model of familial ALS. PLoS ONE 2, e689 (2007).
149. Franz, C. et al. Intraspinal cord delivery of IGF-I mediated by adeno-associated virus 2 is neuroprotective in a rat model of familial ALS. Neurobiol. Dis. 3, 473-481 (2009).
150. Kaspar, B. K., Llado, J., Sherkat, N., Rothstein, J. D. & Gage, F. H. Retrograde viral delivery of IGF-1 prolongs survival in a mouse ALS model. Science 5634, 839-842 (2003). (Pubitemid 36962626)
151. Smith, R. A. et al. Antisense oligonucleotide therapy for neurodegenerative disease. J. Clin. Invest. 8, 2290-2296 (2006). (Pubitemid 44162339)
152. Kanter-Schlifke, I., Georgievska, B., Kirik, D. & Kokaia, M. Seizure suppression by GDNF gene therapy in animal models of epilepsy. Mol. Ther. 15, 1106-1113 (2007). (Pubitemid 46813613)
153. Theofilas, P. et al. Adenosine kinase as a target for therapeutic antisense strategies in epilepsy. Epilepsia 52, 589-601 (2011).
154. Laing, J. M. et al. Intranasal administration of the growth-compromised HSV-2 vector ΔRR prevents kainate-induced seizures and neuronal loss in rats and mice. Mol. Ther. 13, 870-881 (2006).
155. Smitt, P. S., Driesse, M., Wolbers, J., Kros, M. & Avezaat, C. Treatment of relapsed malignant glioma with an adenoviral vector containing the herpes simplex thymidine kinase gene followed by ganciclovir. Mol. Ther. 7, 851-858 (2003). (Pubitemid 36790262)