Parkinson's Disease - CERE-120 (AAV2-neurturin)
• Clinical Studies
J. Siffert, M. A. Stacy, D. Turner, M. Tagliati, R. Alterman, S.A. Factor, N. Boulis
and C. W. Olanow, A.E. Lang, A.M. Lozano and R.T. Bartus
Safety and Feasibility of Intranigral and Intraputaminal CERE-120 (AAV2-neurturin)
administration
in Parkinson's Disease
2011 American Academy of Neurology (AAN) 63rd Annual Meeting
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Travis B. Lewis, BA and David G. Standaert, MD, PhD
Parkinson’s Disease, Primates, and Gene Therapy: Vive la Differénce?
Movement Disorders, Vol. 26, No.1, 2011
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Raymond T. Bartus, PhD, Christopher D. Herzog, PhD, Yaping Chu, PhD, Alistair Wilson, BS, Lamar Brown, BS, Joao Siffert, MD, Eugene M. Johnson, Jr., PhD, C. Warren Olanow, MD, Elliott J. Mufson, PhD, and Jeffrey H. Kordower, PhD.
Bioactivity of AAV2-Neurturin Gene Therapy (CERE-120): Differences Between Parkinson’s Disease and Nonhuman Primate Brains
Movement Disorders, Vol. 26, No.1, 2011
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Alim Louis Benabid
Reflection and Reaction. Gene therapy for Parkinson's disease: do we have the cure?
Lancet Neurology, Vol. 9 December, 2010
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Marks WJ, Bartus RT, Siffert J, Davis CS, Lozano A, Boulis N, Vitek J, Stacy M, Turner D, Verhagen L, Bakay R, Watts R, Guthrie B, Jankovic J, Simpson R, Tagliati M, Alterman R, Stern M, Baltuch G, Starr PA, Larson PS, Ostrem JL, Nutt J, Kieburtz K, Kordower JH, Olanow CW.
Gene delivery of AAV2-neurturin for Parkinson’s disease: a double-blind, randomized, controlled trial.
Lancet Neurology, 2010; 9: 1164-72
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Siffert J., Marks W. J., Jr., Starr P. A., Stacy M. A., Boulis N. M., Vitek J. L., Verhagen L., Watts R. L., Jankovic J., Tagliati M., Stern M. B., Nutt J. G., Kordower J. H., Olanow C. W., Bartus R. T.;
AAV2-neurturin (CERE-120) for advanced Parkinson's disease (PD): Efficacy and safety results from a controlled Phase 2 clinical trial.
Society for Neuroscience, Chicago, IL, 2009
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Bartus R.T., Herzog C.D., Wilson A., Brown L., Chu Y., Siffert J., Mufson E.J., Kordower J.H.
Bioactivity of CERE-120 (AAV2-neurturin gene therapy) in Advanced Parkinson's Disease (PD): Comparison of Post-Mortem and Non-human Primate Brain.
XVIII WFN World Congress on Parkinson's Disease and Related Disorders, 2009
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Marks W.J., Ostrem J.L., Verhagen L., Starr P.A., Larson P.S., Bakay R.A.E., Taylor R., Cahn-Weiner D.A., Stoessl A.J., Olanow C.W., Bartus R.T.
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 Neurology, May 2008, 7:400-08
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• Review Article
Bartus RT, Herzog CD, Bishop K, Ostrove JM, Tuszynski M, Kordower JH, Gasmi M.
Issues regarding gene therapy products for Parkinson's disease: the development of CERE-120 (AAV-NTN) as one reference point.
Parkinsonism and Related Disorders. 2007;13 Suppl 3:S469-77
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• Nonclinical Studies
Christopher D. Herzog, Kathie M. Bishop, Lamar Brown, Alistair Wilson, Jeffrey H. Kordower, Raymond T. Bartus
Gene transfer provides a practical means for safe, long-term,
targeted delivery of biologically active neurotrophic factor
proteins for neurodegenerative diseases
Drug Delivery and Translational Research, Vol. 1, No. 5, October 2011: 361-382
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Raymond T. Bartus, Lamar Brown, Alistair Wilson, Brian Kruegel, Joao Siffert,
Eugene M. Johnson Jr., Jeffrey H. Kordower, Christopher D. Herzog
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
Neurobiology of Disease, 44(2011) 38-52
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Wilson A., Herzog C. D., Brown L., Kruegel B., Bartus R. T.
Enhanced neuroprotection by neurturin (NRTN) in 6-OHDA rats following combined substantia nigral plus striatal delivery of AAV2-NRTN (CERE-120).
Society for Neuroscience Meeting, 2010
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Herzog C. D., Wilson A., Brown L., Roque J., Quinitio C., Kordower J. H., Bartus R. T.
Expression of neurturin following CERE-120 (AAV2-neurturin) administration to the nonhuman primate striatum: Effect of varying volume, flow rate and method of infusion.
Society for Neuroscience Meeting, 2010
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Bartus R. T., Brown L., Wilson A., Kruegel B., Kordower J.H., Herzog C. D.
Safe and effective AAV2-mediated expression of NRTN in the substantia nigra supports nigral targeting with CERE-120 in Parkinson's disease.
Society for Neuroscience Meeting, 2010
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Herzog CD, Brown L, Gammon D, Kruegel B, Lin R, Wilson A, Bolton A, Printz M, Gasmi M, Bishop KM, Kordower JH, Bartus RT.
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.
Neurosurgery. 2009 64(4):602-12.
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Herzog CD, Dass B, Gasmi M, Bakay R, Stansell JE, Tuszynski M, Bankiewicz K, Chen EY, Chu Y, Bishop K, Kordower JH, Bartus RT.
Transgene expression, bioactivity, and safety of CERE-120 (AAV2-neurturin) following delivery to the monkey striatum.
Molecular Therapy. 2008 16(10):1737-44.
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Herzog C.D., Dass B., Holden J.E., Stansell J., Gasmi M, Tuszynski M. Bartus R.T., Kordower J.H. Striatal delivery of CERE-120, an AAV2 vector encoding human neurturin, enhances activity of the dopaminergic nigrostriatal system in aged monkeys.
Movement Disorders, 2007, 22 1124-1132.
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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 RT.
AAV2-mediated delivery of human neurturin to the rat nigrostriatal system: long-term efficacy and tolerability of CERE-120 for Parkinson's disease.
Neurobiology of Disease, 2007, 27 67-76.
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Gasmi M., Herzog C.D., Brandon E.P., Cunningham J.J., Ramirez G.A., Ketchum E.T., and Bartus R.T.
Striatal delivery of neurturin by CERE-120, an AAV2 vector for the treatment of dopaminergic neuron degeneration in Parkinson's disease.
Molecular Therapy, 2007, 15 62-68.
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Kordower J.H., Herzog C.D., Dass B., Bakay R.A.E., Stansell J., Gasmi M., Bartus R.T.
Delivery of neurturin by AAV2 (CERE-120) mediated gene transfer provides structural and functional neuroprotection and neurorestoration in MPTP-treated monkeys.
Annals of Neurology, 2006, 60 706-715.
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Alzheimer's Disease - CERE-110 (AAV2-NGF)
• Clinical Studies
Zoe Arvanitakis, Mark H. Tuszynski, Steven Potkin, Raymond Bartus, David Bennett.
A Phase 1 Clinical Trial of CERE-110 (AAV-NGF ) Gene Delivery in Alzheimer's Disease.
American Academy of Neurology Annual Meeting, 2007
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• Nonlinical Studies
Bishop KM, Hofer EK, Mehta A, Ramirez A, Sun L, Tuszynski M, Bartus RT.
Therapeutic potential of CERE-110 (AAV2-NGF): targeted, stable, and sustained NGF delivery and trophic activity on rodent basal forebrain cholinergic neurons.
Experimental Neurology. 2008 211(2):574-84
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Bishop K. M., Herzog C. D., Zeller J. R., Printz M. A., Gasmi M., Brandon E. P., Boyd R. B., Bartus R. T.
A 2-year study in monkeys to evaluate expression, bioactivity, and safety/tolerability of CERE-110, an AAV2-based vector encoding NGF for the potential treatment of Alzheimer's disease.
Society for Neuroscience, 2006
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Huntington's Disease - CERE-120 (AAV2-neurturin)
• Nonlinical Studies
Ramaswamy S., McBride JL, Han I, Berry-Kravis EM, Zhou L, Herzog CD, Gasmi M, Bartus RT, Kordower JH.
Intrastriatal CERE-120 (AAV-Neurturin) protects striatal and cortical neurons and delays motor deficits in a transgenic mouse model of Huntington's disease.
Neurobiology of Disease. 2009 34(1):40-50.
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Ramaswamy S., McBride J., Herzog C.D., Brandon E., Gasmi M., Bartus R.T., and Kordower J.H.
Neurturin gene therapy improves motor function and prevents death of striatal neurons in a 3-nitropropionic acid rat model of Huntington's disease.
Neurobiology of Disease, 2007, 26 375-84.
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McBride J.L., Ramaswamy S., Gasmi M., Bartus R.T., Herzog C.D., Brandon E.P., Zhou L., Pitzer M.R., Berry-Kravis E.M. and Kordower J.H.
Viral delivery of glial cell line-derived neurotrophic factor improves behavior and protects striatal neurons in a mouse model of Huntington's disease.
Proceedings of the National Academy of Sciences, 2006, 103 9345-50.
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