Transgene Expression, Bioactivity, and Safety of CERE-120 (AAV2-Neurturin) Following Delivery to the Monkey Striatum

Unbiased stereological estimation of the total number of neurons in the subdivisions of the rat hippocampus using the optical fractionator

West, M. J., Slomianka, L., & Gundersen, H. J. G. (1991) · The Anatomical Record · doi:10.1002/ar.1092310411 · Cited by 2,994 · see citing works

Levodopa and the Progression of Parkinson's Disease

The Parkinson Study Group (2004) · New England Journal of Medicine · doi:10.1056/nejmoa033447 · Cited by 1,844 · see citing works

Direct brain infusion of glial cell line–derived neurotrophic factor in Parkinson disease

Gill, S. S., Patel, N. K., Hotton, G. R., O'Sullivan, K., McCarter, R., Bunnage, M., Brooks, D. J., Svendsen, C. N., & Heywood, P. (2003) · Nature Medicine · doi:10.1038/nm850 · Cited by 1,290 · see citing works

Neurodegeneration Prevented by Lentiviral Vector Delivery of GDNF in Primate Models of Parkinson's Disease

Kordower, J. H., Emborg, M. E., Bloch, J., Ma, S. Y., Chu, Y., Leventhal, L., McBride, J. L., Chen, E., Palfi, S., Roitberg, B. Z., et al. (2000) · Science · doi:10.1126/science.290.5492.767 · Cited by 1,244 · see citing works

Randomized controlled trial of intraputamenal glial cell line–derived neurotrophic factor infusion in Parkinson disease

Lang, A. E., Gill, S., Patel, N., Lozano, A., Nutt, J. G., Penn, R., Brooks, D. J., Hotton, G. R., Moro, E., Heywood, P., et al. (2006) · Annals of Neurology · doi:10.1002/ana.20737 · Cited by 987 · see citing works

Functional recovery in parkinsonian monkeys treated with GDNF

Gash, D. M., Zhang, Z., Ovadia, A., Cass, W. A., Yi, A., Simmerman, L., Russell, D., Martin, D., Lapchak, P. A., Collins, F., et al. (1996) · Nature · doi:10.1038/380252a0 · Cited by 955 · see citing works

Randomized, double-blind trial of glial cell line-derived neurotrophic factor (GDNF) in PD

Nutt, J. G., Burchiel, K. J., Comella, C., Jankovic, J. J., Lang, A. E., Laws, E. R., Lozano, A. M., Penn, R. D., Simpson, R. K., Stacy, M. A., et al. (2003) · Neurology · doi:10.1212/wnl.60.1.69 · Cited by 816 · see citing works

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

Marks, W. J., Ostrem, J. L., Verhagen, L., Starr, P. A., Larson, P. S., Bakay, R. A., Taylor, R., Cahn-Weiner, D. A., Stoessl, A. J., Olanow, C. W., et al. (2008) · The Lancet Neurology · doi:10.1016/s1474-4422(08)70065-6 · Cited by 596 · see citing works

Improvement of bilateral motor functions in patients with Parkinson disease through the unilateral intraputaminal infusion of glial cell line—derived neurotrophic factor

Slevin, J. T., Gerhardt, G. A., Smith, C. D., Gash, D. M., Kryscio, R. J., & Young, B. A. (2005) · Journal of neurosurgery · doi:10.3171/jns.2005.102.2.0216 · Cited by 474 · see citing works

Age-associated increases of α-synuclein in monkeys and humans are associated with nigrostriatal dopamine depletion: Is this the target for Parkinson's disease?

Chu, Y., & Kordower, J. H. (2006) · Neurobiology of Disease · doi:10.1016/j.nbd.2006.08.021 · Cited by 396 · see citing works

Ret-dependent and -independent Mechanisms of Glial Cell Line-derived Neurotrophic Factor Signaling in Neuronal Cells*

Trupp, M., Scott, R., Whittemore, S., & Ibáñez, C. (1999) · Journal of Biological Chemistry · doi:10.1074/jbc.274.30.20885 · Cited by 333 · see citing works

Point source concentration of GDNF may explain failure of phase II clinical trial

Salvatore, M. F., Ai, Y., FISCHER, B., ZHANG, A., Grondin, R., Zhang, Z., Gerhardt, G. A., & Gash, D. M. (2006) · Experimental Neurology · doi:10.1016/j.expneurol.2006.07.015 · Cited by 261 · see citing works

Delivery of neurturin by AAV2 (CERE‐120)‐mediated gene transfer provides structural and functional neuroprotection and neurorestoration in MPTP‐treated monkeys

Kordower, J. H., Herzog, C. D., Dass, B., Bakay, R. A. E., Stansell, J., Gasmi, M., & Bartus, R. T. (2006) · Annals of Neurology · doi:10.1002/ana.21032 · Cited by 251 · see citing works

Crossroads in GDNF therapy for Parkinson's disease

Sherer, T., Fiske, B. K., Svendsen, C. N., Lang, A. E., & Langston, J. W. (2006) · Movement Disorders · doi:10.1002/mds.20861 · Cited by 172 · see citing works

Delayed delivery of AAV-GDNF prevents nigral neurodegeneration and promotes functional recovery in a rat model of Parkinson's disease

Wang, L., Muramatsu, S., Lu, Y., Ikeguchi, K., Fujimoto, K., Okada, T., Mizukami, H., Hanazono, Y., Kume, A., Urano, F., et al. (2002) · Gene Therapy · doi:10.1038/sj.gt.3301682 · Cited by 160 · see citing works

Striatal Delivery of Neurturin by CERE-120, an AAV2 Vector for the Treatment of Dopaminergic Neuron Degeneration in Parkinson's Disease

Gasmi, M., Herzog, C. D., Brandon, E. P., Cunningham, J. J., Ramirez, G. A., Ketchum, E. T., & Bartus, R. T. (2006) · Molecular Therapy · doi:10.1038/sj.mt.6300010 · Cited by 153 · see citing works

Neuroprotection by transforming growth factor-β1 involves activation of nuclear factor-κB through phosphatidylinositol-3-OH kinase/Akt and mitogen-activated protein kinase-extracellular-signal regulated kinase1,2 signaling pathways

Zhu, Y., Culmsee, C., Klumpp, S., & Krieglstein, J. (2004) · Neuroscience · doi:10.1016/j.neuroscience.2003.10.037 · Cited by 149 · see citing works

Intraputamenal infusion of GDNF in aged rhesus monkeys: Distribution and dopaminergic effects

Ai, Y., Markesbery, W. R., Zhang, Z., Grondin, R., Elseberry, D., Gerhardt, G. A., & Gash, D. M. (2003) · The Journal of Comparative Neurology · doi:10.1002/cne.10689 · Cited by 149 · see citing works

751. Delayed Delivery of AAV-GDNF Prevents Nigral Neurodegeneration and Promotes Functional Recovery in a Rat Model of Parkinson's Disease

(2002) · Molecular Therapy · doi:10.1016/s1525-0016(16)43581-1 · Cited by 147 · see citing works

AAV2-mediated delivery of human neurturin to the rat nigrostriatal system: Long-term efficacy and tolerability of CERE-120 for Parkinson’s disease

Gasmi, M., Brandon, E. P., Herzog, C. D., Wilson, A., Bishop, K. M., Hofer, E. K., Cunningham, J. J., Printz, M. A., Kordower, J. H., & Bartus, R. T. (2007) · Neurobiology of Disease · doi:10.1016/j.nbd.2007.04.003 · Cited by 141 · see citing works

Striatal delivery of CERE‐120, an AAV2 vector encoding human neurturin, enhances activity of the dopaminergic nigrostriatal system in aged monkeys

Herzog, C. D., Dass, B., Holden, J. E., Stansell, J., Gasmi, M., Tuszynski, M. H., Bartus, R. T., & Kordower, J. H. (2007) · Movement Disorders · doi:10.1002/mds.21503 · Cited by 137 · see citing works

Glial Cell Line-Derived Neurotrophic Factor Increases Stimulus-Evoked Dopamine Release and Motor Speed in Aged Rhesus Monkeys

Grondin, R., Cass, W. A., Zhang, Z., Stanford, J. A., Gash, D. M., & Gerhardt, G. A. (2003) · Journal of Neuroscience · doi:10.1523/jneurosci.23-05-01974.2003 · Cited by 134 · see citing works

Transgenic Activation of Ras in Neurons Promotes Hypertrophy and Protects from Lesion-Induced Degeneration

Heumann, R., Goemans, C. G., Bartsch, D., Lingenhöhl, K., Waldmeier, P. C., Hengerer, B., ALLEGRINI, P. R., Schellander, K., Wagner, E. F., Arendt, T., et al. (2000) · The Journal of Cell Biology · doi:10.1083/jcb.151.7.1537 · Cited by 127 · see citing works

Convection-Enhanced Delivery of Adeno-Associated Virus Type 2 (AAV2) into the Striatum and Transport of AAV2 Within Monkey Brain

Hadaczek, P., Kohutnicka, M., Krauze, M. T., Bringas, J., Pivirotto, P., Cunningham, J., & Bankiewicz, K. S. (2006) · Human Gene Therapy · doi:10.1089/hum.2006.17.291 · Cited by 118 · see citing works

Delivery of a GDNF Gene into the Substantia Nigra after a Progressive 6-OHDA Lesion Maintains Functional Nigrostriatal Connections

Kozlowski, D. A., Connor, B. J., Tillerson, J. L., Schallert, T. J., & Bohn, M. C. (2000) · Experimental Neurology · doi:10.1006/exnr.2000.7463 · Cited by 108 · see citing works

Reprint: Six-Month Continuous Intraputamenal Infusion Toxicity Study of Recombinant Methionyl Human Glial Cell Line-Derived Neurotrophic Factor (r-metHuGDNF) in Rhesus Monkeys

Hovland, D. N., Boyd, R. B., Butt, M. T., Engelhardt, J. A., Moxness, M. S., Ma, M. H., Emery, M. G., Ernst, N. B., Reed, R. P., Zeller, J. R., et al. (2007) · Toxicologic Pathology · doi:10.1177/01926230701481899 · Cited by 108 · see citing works

Dopaminergic neuroprotection and regeneration by neurturin assessed by using behavioral, biochemical and histochemical measurements in a model of progressive Parkinson’s disease

Oiwa, Y., Yoshimura, R., Nakai, K., & Itakura, T. (2002) · Brain Research · doi:10.1016/s0006-8993(02)02934-7 · Cited by 105 · see citing works

Recombinant adeno-associated viral vector (rAAV) delivery of GDNF provides protection against 6-OHDA lesion in the common marmoset monkey (Callithrix jacchus)

Eslamboli, A., Cummings, R. M., Ridley, R. M., Baker, H. F., Muzyczka, N., Bürger, C., Mandel, R. J., Kirik, D., & Annett, L. E. (2003) · Experimental Neurology · doi:10.1016/j.expneurol.2003.08.007 · Cited by 103 · see citing works

Effects of chronic intraputamenal infusion of glial cell line-derived neurotrophic factor (GDNF) in aged Rhesus monkeys

Maswood, N., Grondin, R., Zhang, Z., Stanford, J. A., Surgener, S. P., Gash, D. M., & Gerhardt, G. A. (2002) · Neurobiology of Aging · doi:10.1016/s0197-4580(02)00022-2 · Cited by 70 · see citing works

Synergistic Induction of Neurite Outgrowth by Nerve Growth Factor or Epidermal Growth Factor and Interleukin-6 in PC12 Cells

Wu, Y. Y., & Bradshaw, R. A. (1996) · Journal of Biological Chemistry · doi:10.1074/jbc.271.22.13033 · Cited by 69 · see citing works

Convective delivery of glial cell line–derived neurotrophic factor in the human putamen

Morrison, P. F., Lonser, R. R., & Oldfield, E. H. (2007) · Journal of neurosurgery · doi:10.3171/jns-07/07/0074 · Cited by 69 · see citing works

Hyperplastic Changes within the Leptomeninges of the Rat and Monkey in Response to Chronic Intracerebroventricular Infusion of Nerve Growth Factor

Day-Lollini, P. A., Stewart, G. R., Taylor, M. J., Johnson, R. M., & Chellman, G. J. (1997) · Experimental Neurology · doi:10.1006/exnr.1997.6448 · Cited by 67 · see citing works

Morphological Effects of Estrogen on Cholinergic NeuronsIn VitroInvolves Activation of Extracellular Signal-Regulated Kinases

Dominguez, R., Jalali, C., & Lacalle, S. D. (2004) · Journal of Neuroscience · doi:10.1523/jneurosci.2586-03.2004 · Cited by 60 · see citing works

Intracranial delivery of proteins and peptides as a therapy for neurodegenerative diseases

Grondin, R., Zhang, Z., Ai, Y., Gash, D. M., & Gerhardt, G. A. (2003) · Birkhäuser Basel eBooks · doi:10.1007/978-3-0348-8049-7_4 · Cited by 43 · see citing works

GDNF in treatment of Parkinson's disease: response to editorial

Penn, R. D., Dalvi, A., Slevin, J. T., Young, B. A., Gash, D. M., Gerhardt, G. A., & Hutchinson, M. (2006) · The Lancet Neurology · doi:10.1016/s1474-4422(06)70360-x · Cited by 38 · see citing works

GDNF in treatment of Parkinson's disease: response to editorial

Lang, A. E., Langston, J. W., Stoessl, A. J., Brodsky, M., Brooks, D. J., DHAWAN, V. M., Elias, W. J., Lozano, A. M., Moro, E., Nutt, J. G., et al. (2006) · The Lancet Neurology · doi:10.1016/s1474-4422(06)70359-3 · Cited by 33 · see citing works

Six-Month Continuous Intraputamenal Infusion Toxicity Study of Recombinant Methionyl Human Glial Cell Line-Derived Neurotrophic Factor (r-metHuGDNF) in Rhesus Monkeys

Hovland, D. N., Boyd, R. B., Butt, M. T., Engelhardt, J. A., Moxness, M. S., Ma, M. H., Emery, M. G., Ernst, N. B., Reed, R. P., Zeller, J. R., et al. (2007) · Toxicologic Pathology · doi:10.1177/01926230701481899a · Cited by 31 · see citing works

Untitled work

Kordower (1999) · doi:10.1002/1531-8249(199909)46:3

Gene delivery of AAV2-neurturin for Parkinson's disease: a double-blind, randomised, controlled trial

Marks, W. J., Bartus, R. T., Siffert, J. O., Davis, C. S., Lozano, A. M., Boulis, N. M., Vitek, J. L., Stacy, M. A., Turner, D. A., Verhagen, L., et al. (2010) · The Lancet Neurology · doi:10.1016/s1474-4422(10)70254-4 · Cited by 685 · see citing works

Gene delivery of neurturin to putamen and substantia nigra in P arkinson disease: A double‐blind, randomized, controlled trial

Olanow, C. W., Bartus, R. T., Baumann, T. L., Factor, S. A., Boulis, N. M., Stacy, M. A., Turner, D. A., Marks, W. J., Larson, P., Starr, P. A., et al. (2015) · Annals of Neurology · doi:10.1002/ana.24436 · Cited by 287 · see citing works

Parkinson's Disease Therapeutics: New Developments and Challenges Since the Introduction of Levodopa

Smith, Y., Wichmann, T., Factor, S. A., & DeLong, M. R. (2011) · Neuropsychopharmacology · doi:10.1038/npp.2011.212 · Cited by 246 · see citing works

Bioactivity of AAV2‐neurturin gene therapy (CERE‐120): Differences between Parkinson's disease and nonhuman primate brains

Bartus, R. T., Herzog, C. D., Chu, Y., Wilson, A., Brown, L., Siffert, J. O., Johnson, E. M., Olanow, C. W., Mufson, E. J., & Kordower, J. H. (2010) · Movement Disorders · doi:10.1002/mds.23442 · Cited by 170 · see citing works

Gene Therapy for Parkinson’s Disease, An Update

Axelsen, T. M., & Woldbye, D. P. D. (2018) · Journal of Parkinson s Disease · doi:10.3233/jpd-181331 · Cited by 175 · see citing works

Parkinson's Disease Gene Therapy: Success by Design Meets Failure by Efficacy

Bartus, R. T., Weinberg, M. S., & Samulski, R. J. (2013) · Molecular Therapy · doi:10.1038/mt.2013.281 · Cited by 165 · see citing works

Neurotrophic factor therapy for Parkinson’s disease

Rangasamy, S. B., Soderstrom, K. E., Bakay, R. A. E., & Kordower, J. H. (2010) · Progress in brain research · doi:10.1016/s0079-6123(10)84013-0 · Cited by 155 · see citing works

Comparative Transduction Efficiency of AAV Vector Serotypes 1–6 in the Substantia Nigra and Striatum of the Primate Brain

Markakis, E. A., Vives, K. P., Bober, J., Leichtle, S. W., Léránth, C., Beecham, J., Elsworth, J. D., Roth, R. H., Samulski, R. J., & Redmond, D. E. (2009) · Molecular Therapy · doi:10.1038/mt.2009.286 · Cited by 130 · see citing works

Trophic Factor Gene Therapy for Parkinson's Disease

Kordower, J. H., & Björklund, A. (2013) · Movement Disorders · doi:10.1002/mds.25344 · Cited by 129 · see citing works

Repairing the parkinsonian brain with neurotrophic factors

Aron, L., & Klein, R. (2010) · Trends in Neurosciences · doi:10.1016/j.tins.2010.11.001 · Cited by 126 · see citing works

Viral vectors for neurotrophic factor delivery: A gene therapy approach for neurodegenerative diseases of the CNS

Seung Taik, L. I. M., Airavaara, M., & Harvey, B. K. (2009) · Pharmacological Research · doi:10.1016/j.phrs.2009.10.002 · Cited by 126 · see citing works

Clinical tests of neurotrophic factors for human neurodegenerative diseases, part 1: Where have we been and what have we learned?

Bartus, R. T., & Johnson, E. M. (2017) · Neurobiology of Disease · doi:10.1016/j.nbd.2016.03.027 · Cited by 97 · see citing works

Old and new challenges in Parkinson's disease therapeutics

Pires, A. O., Teixeira, F. G., Mendes-Pinheiro, B., Serra, S. C., Sousa, N., & Salgado, A. J. (2017) · Progress in Neurobiology · doi:10.1016/j.pneurobio.2017.04.006 · Cited by 94 · see citing works

Long-Term Expression and Safety of Administration of AAVrh.10hCLN2 to the Brain of Rats and Nonhuman Primates for the Treatment of Late Infantile Neuronal Ceroid Lipofuscinosis

Sondhi, D., Johnson, L. K., Purpura, K. P., Monette, S., Souweidane, M. M., Kaplitt, M. G., Kosofsky, B. E., Yohay, K. H., Ballon, D., Dyke, J. P., et al. (2012) · Human Gene Therapy Methods · doi:10.1089/hgtb.2012.120 · Cited by 91 · see citing works

Post-mortem assessment of the short and long-term effects of the trophic factor neurturin in patients with α-synucleinopathies

Bartus, R. T., Kordower, J. H., Johnson, E. M., Brown, L., Kruegel, B. R., Chu, Y., Baumann, T. L., Lang, A. E., Olanow, C. W., & Herzog, C. D. (2015) · Neurobiology of Disease · doi:10.1016/j.nbd.2015.03.023 · Cited by 82 · see citing works

Gene Therapy in Parkinsonʼs Disease

Feng, L., & Maguire‐Zeiss, K. A. (2010) · CNS Drugs · doi:10.2165/11533740-000000000-00000 · Cited by 78 · see citing works

EXPRESSION, BIOACTIVITY, AND SAFETY 1 YEAR AFTER ADENO-ASSOCIATED VIRAL VECTOR TYPE 2–MEDIATED DELIVERY OF NEURTURIN TO THE MONKEY NIGROSTRIATAL SYSTEM SUPPORT CERE-120 FOR PARKINSON'S DISEASE

Herzog, C. D., Brown, L., Gammon, D., Kruegel, B. R., Lin, R., Wilson, A., Bolton, A., Printz, M. A., Gasmi, M., Bishop, K. M., et al. (2009) · Neurosurgery · doi:10.1227/01.neu.0000340682.06068.01 · Cited by 75 · see citing works

Postischemic IGF-1 Gene Transfer Promotes Neurovascular Regeneration after Experimental Stroke

Zhu, W. P., Fan, Y., Hao, Q., Shen, F., Hashimoto, T. N., Yang, G., Gasmi, M., Bartus, R. T., Young, W. L., & Chen, Y. (2009) · Journal of Cerebral Blood Flow & Metabolism · doi:10.1038/jcbfm.2009.75 · Cited by 72 · see citing works

Efficient intracerebral delivery of AAV5 vector encoding human ARSA in non-human primate

Colle, M.-A., Piguet, F., Bertrand, L., Raoul, S., Bièche, I., Dubreil, L., Sloothaak, D., Bouquet, C., Moullier, P., Aubourg, P. R., et al. (2009) · Human Molecular Genetics · doi:10.1093/hmg/ddp475 · Cited by 72 · see citing works

Application of CRISPR/Cas9 in Alzheimer’s Disease

Lu, L., Xi, Y., Cai, Y., Sun, M., & Yang, H. (2021) · Frontiers in Neuroscience · doi:10.3389/fnins.2021.803894 · Cited by 78 · see citing works

Clinical tests of neurotrophic factors for human neurodegenerative diseases, part 2: Where do we stand and where must we go next?

Bartus, R. T., & Johnson, E. M. (2016) · Neurobiology of Disease · doi:10.1016/j.nbd.2016.03.026 · Cited by 71 · see citing works

Glial Cell Line-Derived Neurotrophic Factor Gene Delivery in Parkinson's Disease: A Delicate Balance between Neuroprotection, Trophic Effects, and Unwanted Compensatory Mechanisms

Tenenbaum, L., & Humbert‐Claude, M. (2017) · Frontiers in Neuroanatomy · doi:10.3389/fnana.2017.00029 · Cited by 72 · see citing works

Advancing neurotrophic factors as treatments for age-related neurodegenerative diseases: developing and demonstrating “clinical proof-of-concept” for AAV-neurturin (CERE-120) in Parkinson's disease

Bartus, R. T., Baumann, T. L., Brown, L., Kruegel, B. R., Ostrove, J. M., & Herzog, C. D. (2012) · Neurobiology of Aging · doi:10.1016/j.neurobiolaging.2012.07.018 · Cited by 70 · see citing works

Trophic factors for Parkinson's disease: To live or let die

Olanow, C. W., Bartus, R. T., Volpicelli‐Daley, L. A., & Kordower, J. H. (2015) · Movement Disorders · doi:10.1002/mds.26426 · Cited by 66 · see citing works

AAV Vector-Mediated Gene Delivery to Substantia Nigra Dopamine Neurons: Implications for Gene Therapy and Disease Models

Albert, K., Voutilainen, M. H., Domanskyi, A., & Airavaara, M. (2017) · Genes · doi:10.3390/genes8020063 · Cited by 66 · see citing works

Nonhuman Primate Models of Neurodegenerative Disorders

Emborg, M. E. (2017) · ILAR Journal · doi:10.1093/ilar/ilx021 · Cited by 64 · see citing works

Properly scaled and targeted AAV2-NRTN (neurturin) to the substantia nigra is safe, effective and causes no weight loss: Support for nigral targeting in Parkinson's disease

Bartus, R. T., Brown, L., Wilson, A., Kruegel, B. R., Siffert, J. O., Johnson, E. M., Kordower, J. H., & Herzog, C. D. (2011) · Neurobiology of Disease · doi:10.1016/j.nbd.2011.05.026 · Cited by 63 · see citing works

Novel oligodendroglial alpha synuclein viral vector models of multiple system atrophy: studies in rodents and nonhuman primates

Mandel, R. J., Marmion, D. J., Kirik, D., Chu, Y., Heindel, C., McCown, T., Gray, S. J., & Kordower, J. H. (2017) · Acta Neuropathologica Communications · doi:10.1186/s40478-017-0451-7 · Cited by 56 · see citing works

Gene therapy for Parkinson's disease: Disease modification by GDNF family of ligands

Kirik, D., Cederfjäll, E. A., Halliday, G. M., & Petersén, Å. (2016) · Neurobiology of Disease · doi:10.1016/j.nbd.2016.09.008 · Cited by 56 · see citing works

Future of Cell and Gene Therapies for Parkinson’s Disease

Isacson, O., & Kordower, J. (2008) · Annals of Neurology · doi:10.1002/ana.21473 · Cited by 52 · see citing works

AAV viral vector delivery to the brain by shape-conforming MR-guided infusions

Bankiewicz, K. S., Sudhakar, V. R., Samaranch, L., Sebastián, W. S., Bringas, J., & Forsayeth, J. (2016) · Journal of Controlled Release · doi:10.1016/j.jconrel.2016.02.034 · Cited by 52 · see citing works

An optogenetic approach in epilepsy

Kokaia, M., Andersson, M., & Ledri, M. (2012) · Neuropharmacology · doi:10.1016/j.neuropharm.2012.05.049 · Cited by 51 · see citing works

Cell transplantation and gene therapy in Parkinson's disease.

Wakeman, D. R., Dodiya, H. B., & Kordower, J. (2011) · The Mount Sinai journal of medicine, New York · doi:10.1002/msj.20233 · Cited by 60 · see citing works

Trophic factors for Parkinson's disease: Where are we and where do we go from here?

Paul, G., & Sullivan, A. M. (2018) · European Journal of Neuroscience · doi:10.1111/ejn.14102 · Cited by 51 · see citing works

Data-driven evolution of neurosurgical gene therapy delivery in Parkinson’s disease

Richardson, R. M., Bankiewicz, K. S., Christine, C. W., Laar, A. D. V., Gross, R. E., Lonser, R. R., Factor, S. A., Kostyk, S. K., Kells, A. P., Ravina, B. M., et al. (2020) · Journal of Neurology Neurosurgery & Psychiatry · doi:10.1136/jnnp-2020-322904 · Cited by 48 · see citing works

Gene Therapy for Neurological Disorders: Challenges and Future Prospects for the Use of Growth Factors for the Treatment of Parkinsons Disease

Manfredsson, F. P., Okun, M. S., & Mandel, R. J. (2009) · Current Gene Therapy · doi:10.2174/156652309789753400 · Cited by 46 · see citing works

Transplantation of Human Neural Stem Cells in a Parkinsonian Model Exerts Neuroprotection via Regulation of the Host Microenvironment

Zuo, F., Bao, X., Sun, X., Wu, J., Bai, Q., Chen, G., Li, X., Zhou, Q., Yang, Y., Shen, Q., et al. (2015) · International Journal of Molecular Sciences · doi:10.3390/ijms161125966 · Cited by 46 · see citing works

A dual-hit animal model for age-related parkinsonism

Boger, H. A., Granholm, A., McGinty, J. F., & Middaugh, L. D. (2009) · Progress in Neurobiology · doi:10.1016/j.pneurobio.2009.10.013 · Cited by 43 · see citing works

The glial response to intracerebrally delivered therapies for neurodegenerative disorders: is this a critical issue?

Cicchetti, F., & Barker, R. A. (2014) · Frontiers in Pharmacology · doi:10.3389/fphar.2014.00139 · Cited by 39 · see citing works

Scientific rationale for the development of gene therapy strategies for Parkinson's disease

Björklund, T., & Kirik, D. (2009) · Biochimica et Biophysica Acta (BBA) - Molecular Basis of Disease · doi:10.1016/j.bbadis.2009.02.009 · Cited by 37 · see citing works

Viral vector delivery of neurotrophic factors for Parkinson's disease therapy

Kelly, M. J., O’Keeffe, G. W., & Sullivan, A. M. (2015) · Expert Reviews in Molecular Medicine · doi:10.1017/erm.2015.6 · Cited by 36 · see citing works

Non-human primate models of PD to test novel therapies

Morissette, M., & Paolo, T. D. (2017) · Journal of Neural Transmission · doi:10.1007/s00702-017-1722-y · Cited by 35 · see citing works

Controlled dissemination of AAV vectors in the primate brain

Varenika, V., Kells, A. P., Valles, F., Hadaczek, P., Forsayeth, J., & Bankiewicz, K. S. (2009) · Progress in Brain Research · doi:10.1016/s0079-6123(09)17511-8 · Cited by 33 · see citing works

Gene Therapy for Parkinson's Disease

Denyer, R., & Douglas, M. R. (2012) · Parkinson s Disease · doi:10.1155/2012/757305 · Cited by 31 · see citing works

Issues regarding gene therapy products for Parkinson's disease: The development of CERE-120 (AAV-NTN) as one reference point

Bartus, R. T., Herzog, C. D., Bishop, K. M., Ostrove, J. M., Tuszynski, M. H., Kordower, J. H., & Gasmi, M. (2007) · Parkinsonism & Related Disorders · doi:10.1016/s1353-8020(08)70052-x · Cited by 31 · see citing works

Gene transfer provides a practical means for safe, long-term, targeted delivery of biologically active neurotrophic factor proteins for neurodegenerative diseases

Herzog, C. D., Bishop, K. M., Brown, L., Wilson, A., Kordower, J. H., & Bartus, R. T. (2011) · Drug Delivery and Translational Research · doi:10.1007/s13346-011-0037-z · Cited by 29 · see citing works

GDNF signaling in subjects with minimal motor deficits and Parkinson's disease

Chu, Y., & Kordower, J. H. (2021) · Neurobiology of Disease · doi:10.1016/j.nbd.2021.105298 · Cited by 29 · see citing works

Gene Therapy in Movement Disorders: A Systematic Review of Ongoing and Completed Clinical Trials

Merola, A., Kobayashi, N., Romagnolo, A., Wright, B. A., Artusi, C. A., Imbalzano, G., Litvan, I., Laar, A. D. V., & Bankiewicz, K. S. (2021) · Frontiers in Neurology · doi:10.3389/fneur.2021.648532 · Cited by 30 · see citing works

Translating the therapeutic potential of neurotrophic factors to clinical ‘proof of concept’: A personal saga achieving a career-long quest

Bartus, R. T. (2012) · Neurobiology of Disease · doi:10.1016/j.nbd.2012.04.004 · Cited by 28 · see citing works

An Update on Gene Therapy in Parkinson’s Disease

Witt, J. L., & Marks, W. J. (2011) · Current Neurology and Neuroscience Reports · doi:10.1007/s11910-011-0197-8 · Cited by 27 · see citing works

Gene therapy approaches in the non-human primate model of Parkinson’s disease

Pignataro, D., Sucunza, D., Rico, A. J., Dopeso‐Reyes, I. G., Roda, E., Rodriguez-Perez, A. I., Labandeira-García, J. L., Broccoli, V., Kato, S., Kobayashi, K., et al. (2017) · Journal of Neural Transmission · doi:10.1007/s00702-017-1681-3 · Cited by 26 · see citing works

Neurosurgical gene therapy for central nervous system diseases

Patel, R. V., Nanda, P., & Richardson, R. M. (2024) · Neurotherapeutics · doi:10.1016/j.neurot.2024.e00434 · Cited by 23 · see citing works

Gene Therapy for Parkinsons and Alzheimers Diseases: from the Bench to Clinical Trials

Nobre, R. J., & de Almeida, L. P. (2011) · Current Pharmaceutical Design · doi:10.2174/138161211798072472 · Cited by 22 · see citing works

Preclinical Toxicity Evaluation of AAV for Pain: Evidence from Human AAV Studies and from the Pharmacology of Analgesic Drugs

Pleticha, J., Heilmann, L. F., Evans, C. H., Asokan, A., Samulski, R. J., & Beutler, A. S. (2014) · Molecular Pain · doi:10.1186/1744-8069-10-54 · Cited by 21 · see citing works

Viral vector-mediated gene transfer for CNS disease

Snyder, B. R., Boulis, N. M., & Federici, T. (2010) · Expert Opinion on Biological Therapy · doi:10.1517/14712590903514074 · Cited by 20 · see citing works

Novel Gene Therapy Approaches for Targeting Neurodegenerative Disorders: Focusing on Delivering Neurotrophic Genes

Kumari, S., Kamiya, A., Karnik, S. S., Rohilla, S., Dubey, S. K., & Taliyan, R. (2024) · Molecular Neurobiology · doi:10.1007/s12035-024-04260-y · Cited by 19 · see citing works

AAV2-neurturin (CERE-120) for Parkinson's disease

Hickey, P., & Stacy, M. A. (2012) · Expert Opinion on Biological Therapy · doi:10.1517/14712598.2013.754420 · Cited by 18 · see citing works

Overview of Therapeutic Drugs and Methods for the Treatment of Parkinson’s Disease

Schneider, A., Sari, A. T., Alhaddad, H., & Sari, Y. (2020) · CNS & Neurological Disorders - Drug Targets · doi:10.2174/1871527319666200525011110 · Cited by 17 · see citing works

Gene transfer to the rhesus monkey brain using SV40-derived vectors is durable and safe

Louboutin, J. P., Marusich, E., Fisher‐Perkins, J. M., Dufour, J., Bunnell, B. A., & Strayer, D. S. (2011) · Gene Therapy · doi:10.1038/gt.2011.13 · Cited by 17 · see citing works

Focal and dose-dependent neuroprotection in ALS mice following AAV2-neurturin delivery

Gross, S. K., Shim, B. S., Bartus, R. T., Deaver, D., McEachin, Z. T., Bétourné, A., Boulis, N. M., & Maragakis, N. J. (2019) · Experimental Neurology · doi:10.1016/j.expneurol.2019.113091 · Cited by 15 · see citing works

Therapeutic technology

Selzer, M. E., Clarke, S., Cohen, L. G., Kwakkel, G., & Miller, R. H. (2014) · Cambridge University Press eBooks · doi:10.1017/cbo9780511995590.012 · Cited by 12 · see citing works

Gene-based therapy of Parkinson’s Disease: Translation from animal model to human clinical trial employing convection enhanced delivery

Miranpuri, G. S., Kumbier, L., Hinchman, A., Schomberg, D. T., Wang, A., Marshall, H., Kubota, K., Ross, C., & Sillay, K. A. (2012) · Annals of Neurosciences · doi:10.5214/ans.0972.7531.190310 · Cited by 12 · see citing works

Gene Therapy for Parkinson’s Disease Using Midbrain Developmental Genes to Regulate Dopaminergic Neuronal Maintenance

Kim, J., & Chang, M. (2024) · International Journal of Molecular Sciences · doi:10.3390/ijms252212369 · Cited by 10 · see citing works

Bridging between transplantation therapy and neurotrophic factors in Parkinson s disease

Smith, G. M. (2014) · Frontiers in Bioscience-Elite · doi:10.2741/e704 · Cited by 10 · see citing works

Gene therapy for Parkinson’s disease: state-of-the-art treatments for neurodegenerative disease

Douglas, M. R. (2013) · Expert Review of Neurotherapeutics · doi:10.1586/ern.13.58 · Cited by 10 · see citing works

Bone marrow-derived mesenchymal stem cells increase dopamine synthesis in the injured striatum.

Huang, Y. M., Chang, C.-Y., Zhang, J., & Gao, X. (2012) · PubMed Central · doi:10.3969/j.issn.1673-5374.2012.34.002 · Cited by 5 · see citing works

Gene Therapy for Parkinson’s Disease

Douglas, M., & Hazlehurst, J. (2011) · InTech eBooks · doi:10.5772/17420 · Cited by 4 · see citing works

RESTORATION OF MOTOR AND NON-MOTOR FUNCTIONS BY NEUROTROPHIC FACTORS IN NONHUMAN PRIMATES WITH DOPAMINE DEPLETION

Subramanian, K. (2013) · D-Scholarship@Pitt (University of Pittsburgh) · Cited by 2 · see citing works

Transduction profiles in minipig following MRI guided delivery of AAV-5 into thalamic and corona radiata areas

Pietersz, K. L., Pouw, S. M., Klı́ma, J., Ellederová, Z., Bohuslavová, B., Chrastina, J., Liscak, R., Urgošík, D., Stárek, Z., Crha, M., et al. (2021) · Journal of Neuroscience Methods · doi:10.1016/j.jneumeth.2021.109382 · Cited by 1 · see citing works

[Advances in the application of gene therapy for Parkinson's disease with adeno-associated virus].

Chen, Y., Lü, Y., & Li, Z. (2014) · PubMed

Spinal cord injury: mechanisms, cellular and molecular therapies, and human translation

van Niekerk, E. A., & Tuszynski, M. H. (2014) · Cambridge University Press eBooks · doi:10.1017/cbo9780511995590.024

Gene Therapy for Parkinson’s Disease

Wettergren, E. E., Quintino, L., Manfré, G., & Lundberg, C. (2013) · Neuromethods · doi:10.1007/978-1-62703-610-8_10

EXPRESSION, BIOACTIVITY, AND SAFETY 1 YEAR AFTER ADENO-ASSOCIATED VIRAL VECTOR TYPE 2–MEDIATED DELIVERY OF NEURTURIN TO THE MONKEY NIGROSTRIATAL SYSTEM SUPPORT CERE-120 FOR PARKINSON'S DISEASE

Herzog, C. D., Brown, L., Gammon, D., Kruegel, B. R., Lin, R., Wilson, A., Bolton, A., Printz, M. A., Gasmi, M., Bishop, K. M., et al. (2009) · Neurosurgery · doi:10.1227/01.neu.0000340682.06068.01 · Cited by 75 · see citing works

AAV2-mediated delivery of human neurturin to the rat nigrostriatal system: Long-term efficacy and tolerability of CERE-120 for Parkinson’s disease

Gasmi, M., Brandon, E. P., Herzog, C. D., Wilson, A., Bishop, K. M., Hofer, E. K., Cunningham, J. J., Printz, M. A., Kordower, J. H., & Bartus, R. T. (2007) · Neurobiology of Disease · doi:10.1016/j.nbd.2007.04.003 · Cited by 141 · see citing works

Striatal delivery of CERE‐120, an AAV2 vector encoding human neurturin, enhances activity of the dopaminergic nigrostriatal system in aged monkeys

Herzog, C. D., Dass, B., Holden, J. E., Stansell, J., Gasmi, M., Tuszynski, M. H., Bartus, R. T., & Kordower, J. H. (2007) · Movement Disorders · doi:10.1002/mds.21503 · Cited by 137 · see citing works

Striatal Delivery of Neurturin by CERE-120, an AAV2 Vector for the Treatment of Dopaminergic Neuron Degeneration in Parkinson's Disease

Gasmi, M., Herzog, C. D., Brandon, E. P., Cunningham, J. J., Ramirez, G. A., Ketchum, E. T., & Bartus, R. T. (2006) · Molecular Therapy · doi:10.1038/sj.mt.6300010 · Cited by 153 · see citing works

Bioactivity of AAV2‐neurturin gene therapy (CERE‐120): Differences between Parkinson's disease and nonhuman primate brains

Bartus, R. T., Herzog, C. D., Chu, Y., Wilson, A., Brown, L., Siffert, J. O., Johnson, E. M., Olanow, C. W., Mufson, E. J., & Kordower, J. H. (2010) · Movement Disorders · doi:10.1002/mds.23442 · Cited by 170 · see citing works