Eight Years of Clinical Improvement in MPTP-Lesioned Primates After Gene Therapy With AAV2-hAADC
Hadaczek, Piotr, Eberling, Jamie L, Pivirotto, Philip, Bringas, John, Forsayeth, John, Bankiewicz, Krystof S·2010·Molecular Therapy
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- Bioreducible Gene Delivery Platform that Promotes Intracellular Payload Release and Widespread Brain DispersionDivya Rao, Gijung Kwak, Heng Wang, Charles G. Eberhart, Justin S. Hanes, Jung Soo Suk · 2023 · ACS Biomaterials Science & Engineering
- Evaluation of AAV Capsids and Delivery Approaches for Hereditary Hemorrhagic Telangiectasia Gene TherapyAlka Yadav, Rich Liang, Kelly Press, Annika Schmidt, Zahra Shabani, Kun Leng, Calvin Wang, Abinav Sekhar, Joshua Shi, Garth W. Devlin et al. · 2024 · Translational Stroke Research
- Role of Genes and Treatments for Parkinson’s DiseaseFalaq Naz, Yasir Hasan Siddique · 2020 · The Open Biology Journal
- Gene Therapy for Parkinson’s DiseaseMichael Douglas, Jonathan Hazlehurst · 2011 · InTech eBooks
- Disease mechanisms as Subtypes: Mitochondrial and bioenergetic dysfunctionPatricia González‐Rodríguez, Enrico Zampese, Dalton James Surmeier · 2023 · Handbook of clinical neurology
- Assessment of Hippocampal Adeno-Associated Viral Vector Gene Delivery via Frameless Stereotaxis in a Nonhuman PrimateCary H. Leung, Michele Ann Kliem, Brenten L. Heeke, Scott McPhee, Thais Federici, Brooke R. Snyder, Nicholas M. Boulis · 2011 · Stereotactic and Functional Neurosurgery
- Viral vectors in neurodegenerative diseases: immune responses and therapeutic applicationsYifei Wang, Shuqi Mu, Fangxi Liu · 2025 · Frontiers in Neurology
- Updates on the Pathogenesis and Therapeutic Approaches for Hereditary Hemorrhagic TelangiectasiaAlka Y, Zahra Shabani, Jasneet Kaur Dhaliwal, Annika Schmidt, Hua Su · 2025 · Current Vascular Pharmacology
- Regulatory Elements for Gene Therapy of EpilepsyEkaterina Chesnokova, Natalia V. Bal, Ghofran Alhalabi, Pavel M. Balaban · 2025 · Cells
- GDNF and AADC Gene Therapy for Parkinson’s DiseaseKrystof S. Bankiewicz, Waldy San Sebastián, Lluı́s Samaranch, John Forsayeth · 2016 · Springer eBooks
- Gene Delivery and Gene Therapy for Alzheimer’s DiseaseMaya E. Woodbury, Tomomi Kiyota, Tsuneya Ikezu · 2015 · Neuromethods
- Systemic Gene Delivery by Single-Dose Intracardiac Administration of scAAV2/9 and scAAV2/rh10 Variants in Newborn RatsLucie Chansel‐Debordeaux, Mathieu Bourdenx, Nathalie Dutheil, Sandra Doveró, Marie‐Hélène Canron, Clément Jimenez, Erwan Bézard, Benjamin Dehay · 2018 · Human Gene Therapy Methods
- Direct Convective Nervous System Drug Delivery for Patients with Neurodegenerative DisordersRobert Mark Richardson, Paul Larson · 2019 · Elsevier eBooks
- Advances in targeting neurotransmitter systems in dystoniaDora Steel, Kimberley M. Reid, Antonio Pisani, Ellen J. Hess, Susan Helen Fox, Manju A. Kurian · 2023 · International review of neurobiology
- Viral Vectors in Primate Research: Examples from Parkinson’s Disease ResearchGrégory Porras, Benjamin Dehay, Erwan Bézard · 2013 · Neuromethods
- The Use of Viral Vectors to Enhance CognitionTsuneya Ikezu · 2015 · Elsevier eBooks
- Therapeutic Strategies in Neurodegenerative DiseasesAbdulmunaim M. Eid, Alireza Faridar, Stanley H. Appel · 2024 · Springer eBooks
- Efficient transduction of adeno-associated virus vectors into gerbil hippocampus with an appropriate combination of viral capsids and promotersYoshihide Sehara, Kuniko Shimazaki, Fumio Kurosaki, Naoki Kaneko, Ryosuke Uchibori, Masashi Urabe, Kensuke Kawai, Hiroaki Mizukami · 2018 · Neuroscience Letters
- Transduction catalysis: doxorubicin accelerates and enhances rAAV-mediated gene expression in the cortex of mouse, cat and monkeyHongliang Gong, Nini Yuan, Zhiming Shen, Cheng Tang, Stewart D. Shipp, Liling Qian, Yiliang Lu, Ian Max Andolina, Shenghai Zhang, Jihong Wu et al. · 2020 · bioRxiv (Cold Spring Harbor Laboratory)
- Author Response: Safety of AADC Gene Therapy for Moderately Advanced Parkinson Disease: Three-Year Outcomes From the PD-1101 TrialChadwick W. Christine, Robert Mark Richardson, Elisabeth M. Fine, Omar Khwaja, Grace S. Lin Liang, Andreas Meier, Eiry W. Roberts, Krystof S. Bankiewicz, Paul Larson · 2022 · Neurology
- Cell type-selective AADC expression reduces motor deficits and dyskinesia in Parkinson’s disease mouse modelsAnanya Chowdhury, Alex Fraser, Maxwell Departee, Naz Taskin, Meagan A. Quinlan, John K. Mich, Victoria Omstead, Nathaly Lerma, Ximena Opitz-Araya, Alex C. Hughes et al. · 2026 · bioRxiv (Cold Spring Harbor Laboratory)
- CLINICAL DEVELOPMENT OF AAV VECTORSBarrie J. Carter · 2010 · Gene Therapy and Regulation
- Gene therapies for spinocerebellar ataxia type 1Megan Kathryn Keiser · 2013
- Chemogenetics, Induced Neurons and Pluripotent Stem Cells: Towards Advanced Gene and Cell Therapies Targeting EpilepsyNatalia Avaliani · 2015 · Lund University Publications (Lund University)
- [Advances in the application of gene therapy for Parkinson's disease with adeno-associated virus].Yang Chen, Yinghui Lü, Zhaofa Li · 2014 · PubMed
- Clinical Safety and Applications of Stem Cell Gene TherapyCarlo Stephan Jackson, Marco Alessandrini, Michael Sean Pepper · 2017 · Stem cells in clinical applications
- Gene Therapy Approaches for Neurological Lysosomal Storage DiseasesVictoria J. McCurdy · 2014
- RNA interference therapy for the Spinocerebellar ataxiasPavitra S. Ramachandran · 2014
- Gene delivery for Parkinson's diseasePiotr Chmielarz, Anne E. Panhelainen, Mikko Airavaara, Andrii Domanskyi · 2020 · Elsevier eBooks
- Silencing Hyper-Excitable Neurons as a Treatment for Status Epilepticus and Temporal Lobe Epilepsy.Deblina Dey · 2013
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- Gene Therapy for Neurological DiseasesMassimo S. Fiandaca, Shreyas Mahapatra, Suresh de Silva, William J. Bowers, Howard J. Federoff · 2015 · CRC Press eBooks
- NANONEUROLOGYRodica Bălașa · 2015 · Romanian Journal of Neurology
- Gene Therapy For Neurological Disorders: Parkinson’s Disease And GlioblastomaPinshu Zhao, Tori Goertz · 2022 · Journal of Student Research
- Gene therapy under clinical trials for neurodegenerative diseasesBhupender Sahu, Lakshita Tyagi, Prshant Tyagi, Umesh Kumar · 2024 · Elsevier eBooks
- GDNF Gene Therapy for Parkinson’s DiseaseAdrian P. Kells, Amber D. Van Laar, Krystof S. Bankiewicz · 2025 · Springer eBooks
- Recent Therapies Including Gene Therapy and Immunotherapy in Overcoming the Gaps in the Treatment of NeurodegenerationSakshi Goswami, Yeshvandra Verma, Suresh Vir Singh Rana · 2026 · Springer eBooks
- Industry Perspective on Translational and Clinical Pharmacology Aspects of Viral-based Gene Therapies and Vaccines—Key Considerations and Learnings From Approved ProductsHardik R. Mody, Jatin Narula, Prathap Nagaraja Shastri, Dale Miles, Yun Liu, Vittal Shivva, Jimit Girish Raghav, Gillie A. Roth, Anshul Gupta, Bahru A. Habtemariam et al. · 2026 · The AAPS Journal
- The Mechanism of the Parkin-CMA-NLRP3-PANoptosis Signaling Axis in Parkinson's Disease: From Molecular Basis to Therapeutic StrategiesXinghua Tian, Biao Zhang, Yu Wang, Jianshe Wei · 2026 · Progress in Neurobiology
- Alpha-synuclein and neuromelanin converge to promote Parkinson’s disease pathologyAlba Nicolau, Marta González-Sepúlveda, Joana M. Cladera-Sastre, Thais Cuadros, Annabelle Parent, Gerard Roch, Jacopo Marongiu, Benjamin Huynh, Anahid Ansari Mahabadian, Feifei Su et al. · 2026 · bioRxiv (Cold Spring Harbor Laboratory)