Eight Years of Clinical Improvement in MPTP-Lesioned Primates After Gene Therapy With AAV2-hAADC

Gene therapy clinical trials worldwide to 2007—an update

Edelstein, M. L., Abedi, M. R., & Wixon, J. (2007) · Journal of Gene Medicine · doi:10.1002/jgm.1100 · Cited by 629 · 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

Safety and tolerability of putaminal <i>AADC</i> gene therapy for Parkinson disease

Christine, C. W., Starr, P. A., Larson, P. S., Eberling, J. L., Jagust, W. J., Hawkins, R. A., VanBrocklin, H. F., Wright, J. F., Bankiewicz, K. S., & Aminoff, M. J. (2009) · Neurology · doi:10.1212/wnl.0b013e3181c29356 · Cited by 445 · see citing works

Convection-Enhanced Delivery of AAV Vector in Parkinsonian Monkeys; In Vivo Detection of Gene Expression and Restoration of Dopaminergic Function Using Pro-drug Approach

Bankiewicz, K. S., Eberling, J. L., Kohutnicka, M., Jagust, W. J., Pivirotto, P., Bringas, J., Cunningham, J., Budinger, T. F., & Harvey-White, J. D. (2000) · Experimental Neurology · doi:10.1006/exnr.2000.7408 · Cited by 406 · see citing works

Results from a phase I safety trial of <i>hAADC</i> gene therapy for Parkinson disease

Eberling, J. L., Jagust, W. J., Christine, C. W., Starr, P., Larson, P., Bankiewicz, K. S., & Aminoff, M. J. (2008) · Neurology · doi:10.1212/01.wnl.0000312381.29287.ff · Cited by 370 · see citing works

Long-Term Clinical Improvement in MPTP-Lesioned Primates after Gene Therapy with AAV-hAADC

Bankiewicz, K. S., Forsayeth, J., Eberling, J. L., Sánchez‐Pernaute, R., Pivirotto, P., Bringas, J., Herscovitch, P., Carson, R. E., Eckelman, W. C., Reutter, B. W., et al. (2006) · Molecular Therapy · doi:10.1016/j.ymthe.2006.05.005 · Cited by 282 · see citing works

Real-time MR imaging of adeno-associated viral vector delivery to the primate brain

Fiandaca, M. S., Varenika, V., Eberling, J. L., McKnight, T. R., Bringas, J., Pivirotto, P., Beyer, J., Hadaczek, P., Bowers, W. J., Park, J. B., et al. (2008) · NeuroImage · doi:10.1016/j.neuroimage.2008.11.012 · Cited by 122 · see citing works

Safety Evaluation of AAV2-GDNF Gene Transfer into the Dopaminergic Nigrostriatal Pathway in Aged and Parkinsonian Rhesus Monkeys

Su, X., Kells, A. P., Huang, E. J., Lee, H. S., Hadaczek, P., Beyer, J., Bringas, J., Pivirotto, P., Penticuff, J., Eberling, J. L., et al. (2009) · Human Gene Therapy · doi:10.1089/hum.2009.103 · Cited by 112 · see citing works

A Dose-Ranging Study of AAV-hAADC Therapy in Parkinsonian Monkeys

Forsayeth, J., Eberling, J. L., Sanftner, L. M., Zhen, Z., Pivirotto, P., Bringas, J., Cunningham, J., & Bankiewicz, K. S. (2006) · Molecular Therapy · doi:10.1016/j.ymthe.2006.04.008 · Cited by 103 · see citing works

Transduction of Nonhuman Primate Brain with Adeno-Associated Virus Serotype 1: Vector Trafficking and Immune Response

Hadaczek, P., Forsayeth, J., Mirek, H., Munson, K., Bringas, J., Pivirotto, P., L McBride, J., Davidson, B. L., & Bankiewicz, K. S. (2009) · Human Gene Therapy · doi:10.1089/hum.2008.151 · Cited by 90 · see citing works

Striatal Delivery of rAAV-hAADC to Rats with Preexisting Immunity to AAV

Sanftner, L. M., Suzuki, B. M., Doroudchi, M. M., Feng, L., McClelland, A., Forsayeth, J. R., & Cunningham, J. (2004) · Molecular Therapy · doi:10.1016/j.ymthe.2003.12.005 · Cited by 89 · 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

Biodistribution of Adeno-associated Virus Type-2 in Nonhuman Primates after Convection-enhanced Delivery to Brain

Cunningham, J., Pivirotto, P., Bringas, J., Suzuki, B. M., Vijay, S., Sanftner, L. M., Kitamura, M., Chan, C. M., & Bankiewicz, K. S. (2008) · Molecular Therapy · doi:10.1038/mt.2008.111 · Cited by 68 · see citing works

Current status of gene therapy trials for Parkinson's disease

Fiandaca, M. S., Forsayeth, J., & Bankiewicz, K. S. (2007) · Experimental Neurology · doi:10.1016/j.expneurol.2007.08.009 · Cited by 60 · see citing works

Focal striatal dopamine may potentiate dyskinesias in parkinsonian monkeys

Bankiewicz, K. S., Daadi, M. M., Pivirotto, P., Bringas, J., Sanftner, L. M., Cunningham, J., Forsayeth, J., & Eberling, J. L. (2005) · Experimental Neurology · doi:10.1016/j.expneurol.2005.10.022 · Cited by 49 · see citing works

Potential therapeutic uses of BDNF in neurological and psychiatric disorders

Nagahara, A. H., & Tuszynski, M. H. (2011) · Nature Reviews Drug Discovery · doi:10.1038/nrd3366 · Cited by 867 · see citing works

Current prospects for RNA interference-based therapies

Davidson, B. L., & McCray, P. B. (2011) · Nature Reviews Genetics · doi:10.1038/nrg2968 · Cited by 760 · see citing works

State-of-the-art gene-based therapies: the road ahead

Kay, M. A. (2011) · Nature Reviews Genetics · doi:10.1038/nrg2971 · Cited by 672 · see citing works

Neurotoxin-based models of Parkinson's disease

Bové, J., & Perier, C. (2011) · Neuroscience · doi:10.1016/j.neuroscience.2011.10.057 · Cited by 508 · see citing works

The AAV Vector Toolkit: Poised at the Clinical Crossroads

Asokan, A., Schaffer, D. V., & Samulski, R. J. (2012) · Molecular Therapy · doi:10.1038/mt.2011.287 · Cited by 463 · see citing works

Therapeutic AAV Gene Transfer to the Nervous System: A Clinical Reality

Hudry, E., & Vandenberghe, L. H. (2019) · Neuron · doi:10.1016/j.neuron.2019.02.017 · Cited by 368 · see citing works

Long-Term Evaluation of a Phase 1 Study of AADC Gene Therapy for Parkinson's Disease

Mittermeyer, G., Christine, C. W., Rosenbluth, K. H., Baker, S. L., Starr, P. A., Larson, P., Kaplan, P. L., Forsayeth, J., Aminoff, M. J., & Bankiewicz, K. S. (2012) · Human Gene Therapy · doi:10.1089/hum.2011.220 · Cited by 297 · see citing works

Adeno-Associated Virus-Based Gene Therapy for CNS Diseases

Hocquemiller, M., Giersch, L., Audrain, M., Parker, S., & Cartier, N. (2016) · Human Gene Therapy · doi:10.1089/hum.2016.087 · Cited by 291 · see citing works

Nerve Growth Factor Gene Therapy: Activation of Neuronal Responses in Alzheimer Disease

Tuszynski, M. H., Yang, J. H., Barba, D., U, H.-S., Bakay, R. A. E., Pay, M. M., Masliah, E., Conner, J. M., Kobalka, P., Roy, S., et al. (2015) · JAMA Neurology · doi:10.1001/jamaneurol.2015.1807 · Cited by 287 · see citing works

Global CNS Transduction of Adult Mice by Intravenously Delivered rAAVrh.8 and rAAVrh.10 and Nonhuman Primates by rAAVrh.10

Yang, B., Li, S., Wang, H., Guo, Y., Gessler, D. J., Cao, C., Su, Q., Kramer, J., Zhong, L., Ahmed, S. S., et al. (2014) · Molecular Therapy · doi:10.1038/mt.2014.68 · Cited by 213 · see citing works

Gene Therapy for Neurodegenerative Diseases

Sudhakar, V., & Richardson, R. M. (2019) · Neurotherapeutics · doi:10.1007/s13311-018-00694-0 · Cited by 212 · see citing works

Current advances in using neurotrophic factors to treat neurodegenerative disorders

Weissmiller, A. M., & Wu, C. (2012) · Translational Neurodegeneration · doi:10.1186/2047-9158-1-14 · Cited by 211 · see citing works

Gene therapy for the CNS using AAVs: The impact of systemic delivery by AAV9

Saraiva, J., Nobre, R. J., & Pereira de Almeida, L. (2016) · Journal of Controlled Release · doi:10.1016/j.jconrel.2016.09.011 · Cited by 201 · see citing works

Gene therapy for aromatic L-amino acid decarboxylase deficiency by MR-guided direct delivery of AAV2-AADC to midbrain dopaminergic neurons

Pearson, T. S., Gupta, N. M., Sebastián, W. S., Imamura-Ching, J., Viehoever, A. R., Grijalvo-Perez, A., Fay, A. J., Seth, N., Lundy, S. M., Seo, Y., et al. (2021) · Nature Communications · doi:10.1038/s41467-021-24524-8 · Cited by 190 · 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

Magnetic resonance imaging–guided phase 1 trial of putaminal <i>AADC</i> gene therapy for Parkinson's disease

Christine, C. W., Bankiewicz, K. S., Van Laar, A. D., Richardson, R. M., Ravina, B., Kells, A. P., Boot, B., Martin, A. J., Nutt, J., Thompson, M. E., et al. (2019) · Annals of Neurology · doi:10.1002/ana.25450 · Cited by 167 · 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

Persistent Expression of Dopamine-Synthesizing Enzymes 15 Years After Gene Transfer in a Primate Model of Parkinson's Disease

Sehara, Y., Fujimoto, K.-I., Ikeguchi, K., Katakai, Y., Ono, F., Takino, N., Ito, M., Ozawa, K., & Muramatsu, S. (2017) · Human Gene Therapy Clinical Development · doi:10.1089/humc.2017.010 · Cited by 143 · see citing works

Evaluation of Intrathecal Routes of Administration for Adeno-Associated Viral Vectors in Large Animals

Hinderer, C., Bell, P., Katz, N., Vite, C. H., Louboutin, J.-P., Bote, E., Yu, H., Zhu, Y., Casal, M. L., Bagel, J., et al. (2018) · Human Gene Therapy · doi:10.1089/hum.2017.026 · Cited by 141 · see citing works

Development of Intrathecal AAV9 Gene Therapy for Giant Axonal Neuropathy

Bailey, R. M., Armao, D. M., Kalburgi, S. N., & Gray, S. J. (2018) · Molecular Therapy — Methods & Clinical Development · doi:10.1016/j.omtm.2018.02.005 · Cited by 128 · see citing works

Widespread Central Nervous System Gene Transfer and Silencing After Systemic Delivery of Novel AAV-AS Vector

Choudhury, S., Harris, A. F., Cabral, D. J., Keeler, A. M., Sapp, E., Ferreira, J. S., Gray‐Edwards, H. L., Johnson, J. A., Johnson, A. K., Su, Q., et al. (2015) · Molecular Therapy · doi:10.1038/mt.2015.231 · Cited by 110 · see citing works

Clinical Gene Therapy for Neurodegenerative Diseases: Past, Present, and Future

Piguet, F., Alves, S., & Cartier, N. (2017) · Human Gene Therapy · doi:10.1089/hum.2017.160 · Cited by 109 · see citing works

Cerebral infusion of AAV9 vector-encoding non-self proteins can elicit cell-mediated immune responses

Ciesielska, A., Hadaczek, P., Mittermeyer, G., Zhou, S., Wright, J. F., Bankiewicz, K. S., & Forsayeth, J. (2013) · Molecular Therapy · doi:10.1038/mt.2012.167 · Cited by 108 · see citing works

Neurotrophic Factors as Regenerative Therapy for Neurodegenerative Diseases: Current Status, Challenges and Future Perspectives

Ouaamari, Y. E., den Bos, J. V., Willekens, B. M. P., Cools, N., & Wens, I. (2023) · International Journal of Molecular Sciences · doi:10.3390/ijms24043866 · Cited by 107 · see citing works

Gene Therapy for the Nervous System: Challenges and New Strategies

Maguire, C. A., Ramirez, S. H., Merkel, S. F., Sena‐Esteves, M., & Breakefield, X. O. (2014) · Neurotherapeutics · doi:10.1007/s13311-014-0299-5 · Cited by 96 · see citing works

Continuous Dopaminergic Stimulation as a Treatment for Parkinson's Disease: Current Status and Future Opportunities

Olanow, C. W., Calabresi, P., & Obeso, J. A. (2020) · Movement Disorders · doi:10.1002/mds.28215 · Cited by 96 · see citing works

Ly6a Differential Expression in Blood–Brain Barrier Is Responsible for Strain Specific Central Nervous System Transduction Profile of AAV-PHP.B

Batista, A. R., King, O. D., Reardon, C. P., Davis, C., Shankaracharya, Philip, V. M., Gray‐Edwards, H. L., D, N. A. M., Lutz, C. M., Landers, J. E., et al. (2019) · Human Gene Therapy · doi:10.1089/hum.2019.186 · Cited by 94 · see citing works

Characterization of a novel adeno-associated viral vector with preferential oligodendrocyte tropism

Powell, S. K., Khan, N., Parker, C. L., Samulski, R. J., Matsushima, G., Gray, S. J., & McCown, T. J. (2016) · Gene Therapy · doi:10.1038/gt.2016.62 · Cited by 93 · see citing works

Safety of AADC Gene Therapy for Moderately Advanced Parkinson Disease: Three-Year Outcomes From the PD-1101 Trial

Christine, C. W., Richardson, R. M., Van Laar, A. D., Thompson, M. E., Fine, E. M., Khwaja, O. S., Li, C., Liang, G. S., Meier, A., Roberts, E. W., et al. (2022) · Neurology · doi:10.1212/wnl.0000000000012952 · Cited by 89 · see citing works

Gene Therapy Approach with an Emphasis on Growth Factors: Theoretical and Clinical Outcomes in Neurodegenerative Diseases

Parambi, D. G. T., Alharbi, K. S., Kumar, R., Harilal, S., Batiha, G. E., Martins, N. A., Hendawy, O. M., Musa, A., Panda, D. S., & Mathew, B. (2021) · Molecular Neurobiology · doi:10.1007/s12035-021-02555-y · Cited by 75 · see citing works

Clinical Applications Involving CNS Gene Transfer

Kantor, B., McCown, T., Leone, P., & Gray, S. J. (2014) · Advances in Genetics · doi:10.1016/b978-0-12-800149-3.00002-0 · Cited by 74 · see citing works

Adeno‐Associated Viral Vectors for Anterograde Axonal Tracing with Fluorescent Proteins in Nontransgenic and Cre Driver Mice

Harris, J. A., Wook Oh, S., & Zeng, H. (2012) · Current Protocols in Neuroscience · doi:10.1002/0471142301.ns0120s59 · Cited by 72 · see citing works

Genetic therapy for the nervous system

Bowers, W. J., Breakefield, X. O., & Sena‐Esteves, M. (2011) · Human Molecular Genetics · doi:10.1093/hmg/ddr110 · Cited by 70 · 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

Systemic Delivery of Tyrosine-Mutant AAV Vectors Results in Robust Transduction of Neurons in Adult Mice

Iida, A., Takino, N., Miyauchi, H., Shimazaki, K., & Muramatsu, S. (2013) · BioMed Research International · doi:10.1155/2013/974819 · Cited by 63 · see citing works

Applications of positron emission tomography in animal models of neurological and neuropsychiatric disorders

Virdee, K., Cumming, P. D., Caprioli, D., Jupp, B., Rominger, A., Aigbirhio, F. I., Fryer, T. D., Riss, P. J., & Dalley, J. W. (2012) · Neuroscience & Biobehavioral Reviews · doi:10.1016/j.neubiorev.2012.01.009 · Cited by 60 · see citing works

Extensive Transduction and Enhanced Spread of a Modified AAV2 Capsid in the Non-human Primate CNS

Naidoo, J., Stanek, L. M., Ohno, K., Trewman, S., Samaranch, L., Hadaczek, P., O’Riordan, C. R., Sullivan, J., Sebastián, W. S., Bringas, J. R., et al. (2018) · Molecular Therapy · doi:10.1016/j.ymthe.2018.07.008 · Cited by 59 · see citing works

Adeno-associated virus vector delivery to the brain: Technology advancements and clinical applications

Ye, D., Chukwu, C., Yang, Y., Hu, Z., & Chen, H. (2024) · Advanced Drug Delivery Reviews · doi:10.1016/j.addr.2024.115363 · Cited by 58 · see citing works

Distinct transduction profiles in the CNS via three injection routes of AAV9 and the application to generation of a neurodegenerative mouse model

Huda, F., Konno, A., Matsuzaki, Y., Goenawan, H., Miyake, K., Shimada, T., & Hirai, H. (2014) · Molecular Therapy — Methods & Clinical Development · doi:10.1038/mtm.2014.32 · Cited by 58 · 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

Using an engineered glutamate-gated chloride channel to silence sensory neurons and treat neuropathic pain at the source

Weir, G. A., Middleton, S. J., Clark, A. J., Daniel, T., Khovanov, N., McMahon, S. B., & Bennett, D. (2017) · Brain · doi:10.1093/brain/awx201 · Cited by 56 · see citing works

Aromatic L‐Amino Acid Decarboxylase Gene Therapy Enhances Levodopa Response in Parkinson's Disease

Nutt, J. G., Curtze, C., Hiller, A., Anderson, S., Larson, P., Laar, A. D. V., Richardson, R. M., Thompson, M. E., Sedkov, A., Leinonen, M., et al. (2020) · Movement Disorders · doi:10.1002/mds.27993 · Cited by 56 · 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

Atoh1 gene therapy in the cochlea for hair cell regeneration

Richardson, R. T., & Atkinson, P. J. (2015) · Expert Opinion on Biological Therapy · doi:10.1517/14712598.2015.1009889 · Cited by 50 · see citing works

Widespread and sustained target engagement in Huntington’s disease minipigs upon intrastriatal microRNA-based gene therapy

Vallès, A., Evers, M. M., Stam, A., Sogorb-González, M., Brouwers, C. C., Vendrell‐Tornero, C., Broekmans, Ş. A., Paerels, L., Klı́ma, J., Bohuslavová, B., et al. (2021) · Science Translational Medicine · doi:10.1126/scitranslmed.abb8920 · Cited by 49 · see citing works

Safety and tolerability of magnetic resonance imaging-guided convection-enhanced delivery of AAV2-hAADC with a novel delivery platform in nonhuman primate striatum

San Sebastian, W., Richardson, R. M., Kells, A. P., Lamarre, C., Bringas, J., Pivirotto, P., Salegio, E. A., DeArmond, S. J., Forsayeth, J., & Bankiewicz, K. S. (2012) · Human Gene Therapy · doi:10.1089/hum.2011.162 · Cited by 48 · 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

Cellular and Molecular Aspects of Parkinson Treatment: Future Therapeutic Perspectives

Jamebozorgi, K., Taghizadeh, E., Rostami, D., Pormasoumi, H., Barreto, G. E., Hayat, S. M. G., & Sahebkar, A. (2018) · Molecular Neurobiology · doi:10.1007/s12035-018-1419-8 · Cited by 45 · see citing works

Targeted Gene Therapy for the Treatment of Heart Failure

Rapti, K., Chaanine, A. H., & Hajjar, R. J. (2011) · Canadian Journal of Cardiology · doi:10.1016/j.cjca.2011.02.005 · Cited by 42 · see citing works

Broad distribution of ataxin 1 silencing in rhesus cerebella for spinocerebellar ataxia type 1 therapy

Keiser, M. S., Kordower, J. H., Gonzalez‐Alegre, P., & Davidson, B. L. (2015) · Brain · doi:10.1093/brain/awv292 · Cited by 41 · 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

Gene and Cell-Based Therapies for Parkinson's Disease: Where Are We?

Buttery, P. C., & Barker, R. A. (2020) · Neurotherapeutics · doi:10.1007/s13311-020-00940-4 · Cited by 39 · see citing works

Single intra-articular injection of adeno-associated virus results in stable and controllable in vivo transgene expression in normal rat knees

Payne, K. A., Lee, H. H., Haleem, A. M., Martins, C., Yuan, Z., Qiao, C., Xiao, X., & Chu, C. R. (2011) · Osteoarthritis and Cartilage · doi:10.1016/j.joca.2011.04.009 · Cited by 39 · see citing works

Widespread AAV1- and AAV2-mediated transgene expression in the nonhuman primate brain: implications for Huntington's disease

Hadaczek, P., Stanek, L. M., Ciesielska, A., Sudhakar, V. R., Samaranch, L., Pivirotto, P., Bringas, J., O’Riordan, C. R., Mastis, B., Sebastián, W. S., et al. (2016) · Molecular Therapy — Methods & Clinical Development · doi:10.1038/mtm.2016.37 · Cited by 37 · see citing works

The Application of Adeno-Associated Viral Vector Gene Therapy to the Treatment of Fragile X Syndrome

Hampson, D. R., Hooper, A. W. M., & Niibori, Y. (2019) · Brain Sciences · doi:10.3390/brainsci9020032 · 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

Electroacoustic Stimulation: Now and into the Future

Irving, S., Gillespie, L. N., Richardson, R. T., Rowe, D., Fallon, J. B., & Wise, A. K. (2014) · BioMed Research International · doi:10.1155/2014/350504 · Cited by 34 · see citing works

Adeno-Associated Viral Vectors as Versatile Tools for Parkinson’s Research, Both for Disease Modeling Purposes and for Therapeutic Uses

Fajardo-Serrano, A., Rico, A. J., Roda, E., Honrubia, A., Arrieta, S., Ariznabarreta, G., Chocarro, J., Lorenzo-Ramos, E., Pejenaute, A., Vázquez, A., et al. (2021) · International Journal of Molecular Sciences · doi:10.3390/ijms22126389 · Cited by 32 · see citing works

Safety and tolerability of MRI-guided infusion of AAV2-hAADC into the mid-brain of nonhuman primate

Sebastián, W. S., Kells, A. P., Bringas, J., Samaranch, L., Hadaczek, P., Ciesielska, A., Macayan, M., Pivirotto, P., Forsayeth, J., Osborne, S., et al. (2014) · Molecular Therapy — Methods & Clinical Development · doi:10.1038/mtm.2014.49 · Cited by 32 · see citing works

A next step in adeno‐associated virus‐mediated gene therapy for neurological diseases: regulation and targeting

Chtarto, A., Bockstael, O., Tshibangu, T., Dewitte, O., Levivier, M., & Tenenbaum, L. (2013) · British Journal of Clinical Pharmacology · doi:10.1111/bcp.12065 · Cited by 32 · see citing works

Gene transfer to the CNS is efficacious in immune-primed mice harboring physiologically relevant titers of anti-AAV antibodies

Treleaven, C. M., Tamsett, T. J., Bu, J., Fidler, J. A., Sardi, S. P., Hurlbut, G. D., Woodworth, L. A., Cheng, S. H., Passini, M. A., Shihabuddin, L. S., et al. (2012) · Molecular Therapy · doi:10.1038/mt.2012.114 · Cited by 31 · 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

Recent Advances in RNA Interference Therapeutics for CNS Diseases

Ramachandran, P. S., Keiser, M. S., & Davidson, B. (2013) · Neurotherapeutics · doi:10.1007/s13311-013-0183-8 · Cited by 30 · 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

Management of Leigh syndrome: Current status and new insights

Chen, L., Cui, Y., Jiang, D., Ma, C. Y., Tse, H., Hwu, W., & Lian, Q. (2017) · Clinical Genetics · doi:10.1111/cge.13139 · Cited by 29 · see citing works

Novel siRNA delivery strategy: a new "strand" in CNS translational medicine?

Gherardini, L., Bardi, G., Gennaro, M., & Pizzorusso, T. (2014) · Cellular and Molecular Life Sciences · doi:10.1007/s00018-013-1310-8 · Cited by 28 · 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

Overexpression of the Steroidogenic Enzyme Cytochrome P450 Side Chain Cleavage in the Ventral Tegmental Area Increases 3α,5α-THP and Reduces Long-Term Operant Ethanol Self-Administration

Cook, J. B., Werner, D. F., Maldonado‐Devincci, A. M., Leonard, M. N., Fisher, K. R., O’Buckley, T. K., Porcu, P., McCown, T. J., Besheer, J., Hodge, C. W., et al. (2014) · Journal of Neuroscience · doi:10.1523/jneurosci.4733-13.2014 · Cited by 28 · see citing works

Cell reprogramming approaches in gene- and cell-based therapies for Parkinson's disease

Man, J. H. K., Groenink, L., & Caiazzo, M. (2018) · Journal of Controlled Release · doi:10.1016/j.jconrel.2018.07.017 · Cited by 26 · see citing works

Delivery of neurotrophic factors in the treatment of age-related chronic neurodegenerative diseases

Padmakumar, S., Taha, M. S., Kadakia, E., Bleier, B. S., & Amiji, M. M. (2020) · Expert Opinion on Drug Delivery · doi:10.1080/17425247.2020.1727443 · Cited by 25 · see citing works

Current prospects and challenges for epilepsy gene therapy

Weinberg, M. S., & McCown, T. J. (2011) · Experimental Neurology · doi:10.1016/j.expneurol.2011.10.003 · Cited by 24 · see citing works

Focusing Brain Therapeutic Interventions in Space and Time for Parkinson’s Disease

Little, S. J., & Brown, P. (2014) · Current Biology · doi:10.1016/j.cub.2014.08.002 · Cited by 22 · see citing works

Changing channels in pain and epilepsy: Exploiting ion channel gene therapy for disorders of neuronal hyperexcitability

Snowball, A., & Schorge, S. (2015) · FEBS Letters · doi:10.1016/j.febslet.2015.05.004 · Cited by 22 · see citing works

Recent developments in nucleic acid-based therapies for Parkinson’s disease: Current status, clinical potential, and future strategies

Pandey, S. K., & Singh, R. K. (2022) · Frontiers in Pharmacology · doi:10.3389/fphar.2022.986668 · Cited by 22 · 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

Challenges for the application of optical stimulation in the cochlea for the study and treatment of hearing loss

Richardson, R. T., Thompson, A. C., Wise, A. K., & Needham, K. (2016) · Expert Opinion on Biological Therapy · doi:10.1080/14712598.2017.1271870 · Cited by 22 · see citing works

Targeting the Central Nervous System with Herpes Simplex Virus / Sleeping Beauty Hybrid Amplicon Vectors

de Silva, S., & J. Bowers, W. (2011) · Current Gene Therapy · doi:10.2174/156652311797415845 · Cited by 20 · see citing works

Advancing gene therapies, methods, and technologies for Parkinson’s Disease and other neurological disorders

Fiandaca, M. S., Lonser, R. R., Elder, J. B., Ząbek, M., & Bankiewicz, K. S. (2020) · Neurologia i Neurochirurgia Polska · doi:10.5603/pjnns.a2020.0046 · Cited by 20 · see citing works

Vector-mediated l-3,4-dihydroxyphenylalanine delivery reverses motor impairments in a primate model of Parkinson’s disease

Rosenblad, C., Li, Q., Pioli, E. Y., Doveró, S., Antunes, A. S. L. M., Agúndez, L., Bardelli, M., Linden, R. M., Henckaerts, E., Björklund, A., et al. (2019) · Brain · doi:10.1093/brain/awz176 · Cited by 20 · see citing works

Gene delivery of a modified antibody to Aβ reduces progression of murine Alzheimer’s disease

Elmer, B. M., Swanson, K. A., Bangari, D. S., Piepenhagen, P. A., Roberts, E., Taksir, T. V., Guo, L. M., Obinu, M. C., Barnéoud, P., Ryan, S., et al. (2019) · PLoS ONE · doi:10.1371/journal.pone.0226245 · Cited by 20 · see citing works

Quantitative, noninvasive, in vivo longitudinal monitoring of gene expression in the brain by co-AAV transduction with a PET reporter gene

Yoon, S. Y., Gay-Antaki, C., Ponde, D. E., Poptani, H., Vite, C. H., & Wolfe, J. H. N. (2014) · Molecular Therapy — Methods & Clinical Development · doi:10.1038/mtm.2014.16 · Cited by 19 · see citing works

Retrograde capabilities of adeno-associated virus vectors in the central nervous system

Surdyka, M., & Figiel, M. (2021) · Biotechnologia · doi:10.5114/bta.2021.111111 · Cited by 19 · see citing works

Adeno-Associated Viral Gene Delivery in Neurodegenerative Disease

Morgenstern, P. F., Marongiu, R., Musatov, S. A., & Kaplitt, M. G. (2011) · Methods in molecular biology · doi:10.1007/978-1-61779-328-8_29 · Cited by 19 · see citing works

Gene therapy to the blood–brain barrier with resulting protein secretion as a strategy for treatment of Niemann Picks type C2 disease

Hede, E., Christiansen, C., Heegaard, C. W., Moos, T., & Burkhart, A. (2020) · Journal of Neurochemistry · doi:10.1111/jnc.14982 · Cited by 19 · see citing works

AAV5-miHTT gene therapy for Huntington disease: lowering both huntingtins

Evers, M. M., & Konstantinova, P. S. (2020) · Expert Opinion on Biological Therapy · doi:10.1080/14712598.2020.1792880 · Cited by 18 · see citing works

Treating hearing disorders with cell and gene therapy

Gillespie, L. N., Richardson, R. T., Nayagam, B. A., & Wise, A. K. (2014) · Journal of Neural Engineering · doi:10.1088/1741-2560/11/6/065001 · Cited by 17 · see citing works

Oscillations in the basal ganglia in Parkinson's disease: Role of the striatum

Belluscio, M., Escande, M. V., Keifman, E., Riquelme, L. A., Murer, M. G., & Zold, C. L. (2013) · Basal Ganglia · doi:10.1016/j.baga.2013.11.003 · Cited by 17 · see citing works

Channelrhodopsins—Their potential in gene therapy for neurological disorders

Ji, Z.-G., Ishizuka, T., & Yawo, H. (2012) · Neuroscience Research · doi:10.1016/j.neures.2012.09.004 · Cited by 15 · see citing works

Transduction catalysis: Doxorubicin amplifies rAAV-mediated gene expression in the cortex of higher-order vertebrates

Gong, H., Yuan, N., Shen, Z., Tang, C., Shipp, S. D., Liling, Q., Lu, Y., Andolina, I. M., Zhang, S., Wu, J., et al. (2021) · iScience · doi:10.1016/j.isci.2021.102685 · Cited by 14 · see citing works

Advances, Nuances, and Potential Pitfalls When Exploiting the Therapeutic Potential of RNA Interference

Battistella, M., & Marsden, P. A. (2015) · Clinical Pharmacology & Therapeutics · doi:10.1002/cpt.8 · Cited by 14 · see citing works

Zinc Finger Repressors mediate widespread PRNP lowering in the nonhuman primate brain and profoundly extend survival in prion disease micePreprint

Chou, S.-W., Mortberg, M. A., Marlen, K., Ojala, D. S., Parman, T., Howard, M., DeSouza-Lenz, K., Lian, Y., Mehrabian, M., Tiffany, M. R., et al. (2025) · bioRxiv (Cold Spring Harbor Laboratory) · doi:10.1101/2025.03.05.636713 · Cited by 14 · see citing works

Non-clinical safety assessment of novel drug modalities: Genome safety perspectives on viral-, nuclease- and nucleotide-based gene therapies

Lemmens, M., Dorsheimer, L., Zeller, A., & Dietz, Y. (2024) · Mutation Research/Genetic Toxicology and Environmental Mutagenesis · doi:10.1016/j.mrgentox.2024.503767 · Cited by 13 · see citing works

Focused Ultrasound-Mediated Disruption of the Blood–Brain Barrier for AAV9 Delivery in a Mouse Model of Huntington’s Disease

Owusu-Yaw, B. S., Zhang, Y., Garrett, L., Yao, A., Shing, K., Batista, A. R., Sena‐Esteves, M., Upadhyay, J., Kegel-Gleason, K. B., & Todd, N. (2024) · Pharmaceutics · doi:10.3390/pharmaceutics16060710 · Cited by 13 · see citing works

Gene therapy for Parkinson’s disease: where are we now and where are we going?

Forsayeth, J., Bankiewicz, K. S., & Aminoff, M. J. (2010) · Expert Review of Neurotherapeutics · doi:10.1586/ern.10.161 · Cited by 13 · see citing works

GABAergic Gene Regulatory Elements Used in Adeno-Associated Viral Vectors

Duba-Kiss, R., Niibori, Y., & Hampson, D. R. (2021) · Frontiers in Neurology · doi:10.3389/fneur.2021.745159 · 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

Nonhuman Primate Models of Huntington’s Disease and Their Application in Translational Research

Badin, R. A. (2018) · Methods in molecular biology · doi:10.1007/978-1-4939-7825-0_14 · Cited by 11 · see citing works

PET imaging in animal models of Parkinson’s disease

Ni, R. (2022) · Behavioural Brain Research · doi:10.1016/j.bbr.2022.114174 · Cited by 11 · see citing works

In utero gene transfer to the mouse nervous system

Rahim, A. A., Wong, A. M. S., Buckley, S. M. K., Chan, J. K. Y., David, A. L., Cooper, J. D., Coutelle, C. C., Peebles, D., & Waddington, S. N. (2010) · Biochemical Society Transactions · doi:10.1042/bst0381489 · Cited by 11 · see citing works

Gene therapy for Parkinson’s disease: a new frontier in neurodegenerative diseases

Asuku, A. O., Ogungbangbe, G. O., & Fajemidagba, G. A. (2025) · Exploration of Neuroprotective Therapy · doi:10.37349/ent.2025.1004119 · Cited by 10 · see citing works

Long-Term Clinical Improvement in MPTP-Lesioned Primates after Gene Therapy with AAV-hAADC

Bankiewicz, K. S., Forsayeth, J., Eberling, J. L., Sánchez‐Pernaute, R., Pivirotto, P., Bringas, J., Herscovitch, P., Carson, R. E., Eckelman, W. C., Reutter, B. W., et al. (2006) · Molecular Therapy · doi:10.1016/j.ymthe.2006.05.005 · Cited by 282 · see citing works

Development of AAV2-hAADC Gene Therapy for Parkinson's Disease designed to Enhance Dopamine Metabolism

Forsayeth, J., & Bankiewicz, K. S. (2008) · Japanese Journal of Neurosurgery · doi:10.7887/jcns.17.323

Persistent Expression of Dopamine-Synthesizing Enzymes 15 Years After Gene Transfer in a Primate Model of Parkinson's Disease

Sehara, Y., Fujimoto, K.-I., Ikeguchi, K., Katakai, Y., Ono, F., Takino, N., Ito, M., Ozawa, K., & Muramatsu, S. (2017) · Human Gene Therapy Clinical Development · doi:10.1089/humc.2017.010 · Cited by 143 · see citing works

Long-Term Evaluation of a Phase 1 Study of AADC Gene Therapy for Parkinson's Disease

Mittermeyer, G., Christine, C. W., Rosenbluth, K. H., Baker, S. L., Starr, P. A., Larson, P., Kaplan, P. L., Forsayeth, J., Aminoff, M. J., & Bankiewicz, K. S. (2012) · Human Gene Therapy · doi:10.1089/hum.2011.220 · Cited by 297 · see citing works

Safety and tolerability of putaminal <i>AADC</i> gene therapy for Parkinson disease

Christine, C. W., Starr, P. A., Larson, P. S., Eberling, J. L., Jagust, W. J., Hawkins, R. A., VanBrocklin, H. F., Wright, J. F., Bankiewicz, K. S., & Aminoff, M. J. (2009) · Neurology · doi:10.1212/wnl.0b013e3181c29356 · Cited by 445 · see citing works