Adeno-Associated Virus-Based Gene Therapy for CNS Diseases

Cited by (290)

Items 101–200 of 290

1. Seven-year follow-up of durability and safety of AAV CNS gene therapy for a lysosomal storage disorder in a large animal
   Sara Marcó, Virginia Haurigot, Maria Luisa Jaén, Albert Ribera, Víctor Sánchez, Maria Molas, Miguel Garcia, Xavier León, Carles Roca, Xavier Sánchez et al. · 2021 · Molecular Therapy — Methods & Clinical Development
2. Recombinant Adeno-Associated Virus Vectors for Gene Therapy of the Central Nervous System: Delivery Routes and Clinical Aspects
   Żaneta Słyk, Natalia Stachowiak, Maciej Małecki · 2024 · Biomedicines
3. Gene-Based Approaches to Inherited Neurometabolic Diseases
   Valentina Poletti, Alessandra Biffi · 2019 · Human Gene Therapy
4. High throughput screening of novel AAV capsids identifies variants for transduction of adult NSCs within the subventricular zone
   Lukas P. M. Kremer, Santiago Cerrizuela, Sascha Dehler, Thomas Stiehl, Jonas Weinmann, Heike Abendroth, Susanne Kleber, Alexander Laure, Jihad El Andari, Simon Anders et al. · 2021 · Molecular Therapy — Methods & Clinical Development
5. Adeno‐associated virus‐based Alzheimer's disease mouse models and potential new therapeutic avenues
   Lars Matthias Ittner, Matthias Klugmann, Yazi Diana Ke · 2019 · British Journal of Pharmacology
6. Gene suppressing therapy for Pelizaeus-Merzbacher disease using artificial microRNA
   Heng Li, Hironori Okada, Sadafumi Suzuki, Kazuhisa Sakai, Hitomi Izumi, Yukiko Matsushima, Noritaka Ichinohe, Yu‐ichi Goto, Takashi Okada, Ken Inoue · 2019 · JCI Insight
7. Gene therapy in epilepsy
   Lu Zhang, Yuping Wang · 2021 · Biomedicine & Pharmacotherapy
8. DREADD Activation of Pedunculopontine Cholinergic Neurons Reverses Motor Deficits and Restores Striatal Dopamine Signaling in Parkinsonian Rats
   Puneet Kumar Sharma, Lisa A. Wells, Gaia Rizzo, Joanna L. Elson, Jan Passchier, Eugenii A. Rabiner, Roger N. Gunn, David Trevor Dexter, Ilse Sanet Pienaar · 2020 · Neurotherapeutics
9. Gene Therapeutic Approaches for the Treatment of Mitochondrial Dysfunction in Parkinson’s Disease
   Jannik Prasuhn, Norbert Brüggemann · 2021 · Genes
10. AAV-mediated neuronal expression of an scFv antibody selective for Aβ oligomers protects synapses and rescues memory in Alzheimer models
    María Clara Sellés, Juliana T.S. Fortuna, Magalí Cecilia Cercato, Luís E. Santos, Luciana Domett, Andre L.B. Bitencourt, Mariane Favero Carraro, Amanda Santos de Souza, Helena Janíčková, Caroline Vieira Azevedo et al. · 2022 · Molecular Therapy
11. In Vivo Gene Therapy for Mucopolysaccharidosis Type III (Sanfilippo Syndrome): A New Treatment Horizon
    Sara Marcó, Virginia Haurigot, Fátima Bosch · 2019 · Human Gene Therapy
12. AAVrh10 Vector Corrects Disease Pathology in MPS IIIA Mice and Achieves Widespread Distribution of SGSH in Large Animal Brains
    Michaël Hocquemiller, Kim M. Hemsley, Meghan L. Douglass, Sarah J. Tamang, Daniel Neumann, Barbara M. King, Helen Beard, Paul J. Trim, Leanne K. Winner, Adeline A. Lau et al. · 2019 · Molecular Therapy — Methods & Clinical Development
13. Intramuscular Delivery of Gene Therapy for Targeting the Nervous System
    Tosolini, Andrew P., Sleigh, James N. · 2020 · Frontiers in Molecular Neuroscience
14. Adeno-Associated Viral Vectors as Versatile Tools for Parkinson’s Research, Both for Disease Modeling Purposes and for Therapeutic Uses
    Fajardo-Serrano, Ana, Rico, Alberto J., Roda, Elvira, Honrubia, Adriana, Arrieta, Sandra, Ariznabarreta, Goiaz, Chocarro, Julia, Lorenzo-Ramos, Elena, Pejenaute, Alvaro, Vázquez, Alfonso et al. · 2021 · International Journal of Molecular Sciences
15. Therapeutic strategies for Huntington's disease
    Carlos Estevez‐Fraga, Michael D. Flower, Sarah J. Tabrizi · 2020 · Current Opinion in Neurology
16. Management of Leigh syndrome: Current status and new insights
    Loretta Chen, Yu Cui, Dan Jiang, C.Y. Ma, Hung‐Fat Tse, Wuh‐Liang Hwu, Qizhou Lian · 2017 · Clinical Genetics
17. CRISPR/Cas9-Based therapeutics as a promising strategy for management of Alzheimer’s disease: progress and prospects
    Mohamad Sultan Khan, Nousheen Qureshi, Rehan Khan, Young‐Ok Son, Tariq Maqbool · 2025 · Frontiers in Cellular Neuroscience
18. DNA‐editing enzymes as potential treatments for heteroplasmic mtDNA diseases
    Ugne Zekonyte, Sandra R. Bacman, Carlos Torres Moraes · 2020 · Journal of Internal Medicine
19. An Improved Adeno-Associated Virus Vector for Neurological Correction of the Mouse Model of Mucopolysaccharidosis IIIA
    Anna L. Gray, Claire O’Leary, Aiyin Liao, Leticia Agúndez, Amir Saam Youshani, Hélène F.E. Gleitz, Helen Parker, Jessica Taylor, Olivier Danos, Michaël Hocquemiller et al. · 2019 · Human Gene Therapy
20. Genetic targeting of astrocytes to combat neurodegenerative disease
    GregoryW Kirschen, Rachel Kéry, AllenP. F. Chen · 2019 · Neural Regeneration Research
21. A comparison of AAV-vector production methods for gene therapy and preclinical assessment
    Marcus Davidsson, Matilde Negrini, Swantje Hauser, Alexander Svanbergsson, Marcus Lockowandt, Giuseppe Tomasello, Fredric P. Manfredsson, Andreas Heuer · 2020 · Scientific Reports
22. Rational design and experimental evaluation of peptide ligands for the purification of adeno‐associated viruses via affinity chromatography
    Shriarjun Shastry, Wenning Chu, Eduardo Barbieri, Paul Greback‐Clarke, William Kojo Smith, Christopher Cummings, Arianna Minzoni, Jennifer Pancorbo, Gary L. Gilleskie, Kimberly Ritola et al. · 2023 · Biotechnology Journal
23. Genome Editing in a Wide Area of the Brain Using Dendrimer-Based Ternary Polyplexes of Cas9 Ribonucleoprotein
    Toru Taharabaru, Ryoma Yokoyama, Taishi Higashi, Ahmed Fouad Abdelwahab Mohammed, Masamichi Inoue, Yuki K. Maeda, Takuro Niidome, Risako Onodera, Keiichi Motoyama · 2020 · ACS Applied Materials & Interfaces
24. miRNA-Mediated Knockdown of ATXN3 Alleviates Molecular Disease Hallmarks in a Mouse Model for Spinocerebellar Ataxia Type 3
    Rui Jorge Nobre, Diana Duarte Lobo, Carina Henriques, Sónia Duarte, Sara Lopes, Ana C. Silva, Miguel Monteiro Lopes, Fanny Mariet, Lukas K. Schwarz, M. S. Baatje et al. · 2021 · Nucleic Acid Therapeutics
25. Neuro-Immuno-Gene- and Genome-Editing-Therapy for Alzheimer’s Disease: Are We There Yet?
    Sudhanshu P. Raikwar, Ramasamy Thangavel, Iuliia Dubova, Mohammad Ejaz Ahmed, Govindhasamy Pushpavathi Selvakumar, Duraisamy Kempuraj, Smita A. Zaheer, Shankar S. Iyer, Asgar Zaheer · 2018 · Journal of Alzheimer s Disease
26. The Influence of Murine Genetic Background in Adeno-Associated Virus Transduction of the Mouse Brain
    Ting He, Michelle S. Itano, Lauriel F. Earley, Nikita Elexa Hall, Natallia V. Riddick, Richard Jude Samulski, Chengwen Li · 2019 · Human Gene Therapy Clinical Development
27. Tight junction modulators for drug delivery to the central nervous system
    Yosuke Hashimoto, Keisuke Tachibana, Masuo Kondoh · 2020 · Drug Discovery Today
28. Granulocyte-colony stimulating factor gene therapy as a novel therapeutics for stroke in a mouse model
    Janet Menzie, Jigar Pravinchandra Modi, Hongyaun Xu, Andrew Bent, Paula Trujillo, Kristen Medley, Eugenia Jimenez, Jessica Shen, Michael Marshall, Rui Tao et al. · 2020 · Journal of Biomedical Science
29. Prenatal treatment path for angelman syndrome and other neurodevelopmental disorders
    Mark J. Zylka · 2019 · Autism Research
30. Intravenous AAV9 administration results in safe and widespread distribution of transgene in the brain of mini-pig
    Yingqi Lin, Caijuan Li, Wei Wang, Jiawei Li, Chunhui Huang, Xiao Zheng, Zhaoming Liu, Xichen Song, Yizhi Chen, Jiale Gao et al. · 2023 · Frontiers in Cell and Developmental Biology
31. Genome Editing in Neurosciences
    Rudolf Jaenisch, Fred H. Gage, Feng Zhang · 2017 · Research and perspectives in neurosciences
32. AAV9-NGLY1 gene replacement therapy improves phenotypic and biomarker endpoints in a rat model of NGLY1 Deficiency
    Lei Zhu, Brandon Tan, Selina S. Dwight, Brendan J. Beahm, Matt Wilsey, Brett E. Crawford, Becky Schweighardt, Jennifer W. Cook, Thomas Wechsler, William F. Mueller · 2022 · Molecular Therapy — Methods & Clinical Development
33. Targeting the central nervous system in lysosomal storage diseases: Strategies to deliver therapeutics across the blood-brain barrier
    Bethan J. Critchley, Hubert Bobby Gaspar, Sara Benedetti · 2022 · Molecular Therapy
34. Dual muscle-liver transduction imposes immune tolerance for muscle transgene engraftment despite preexisting immunity
    Laurent Bartolo, Stéphanie Li Chung Tong, Pascal Chappert, Dominique Urbain, Fanny Collaud, Pasqualina Colella, Isabelle Richard, Giuseppe Ronzitti, Jocelyne Demengeot, David‐Alexandre Gross et al. · 2019 · JCI Insight
35. Revisiting gene delivery to the brain: silencing and editing
    João Conniot, Sepehr Talebian, Susana M. N. Simões, Lino Ferreira, João Conde · 2020 · Biomaterials Science
36. Engineered viral vectors for functional interrogation, deconvolution, and manipulation of neural circuits
    Sabrina Sun, David V. Schaffer · 2018 · Current Opinion in Neurobiology
37. Superior human hepatocyte transduction with adeno-associated virus vector serotype 7
    Shao, Wenwei, Pei, Xiaolei, Cui, Caibin, Askew, Charles, Dobbins, Amanda, Chen, Xiaojing, Abajas, Yasmina L., Gerber, David A., Samulski, R. Jude, Nichols, Timothy C. et al. · 2019 · Gene Therapy
38. An innate immune response to adeno-associated virus genomes decreases cortical dendritic complexity and disrupts synaptic transmission
    Suriano, Christos M., Kumar, Neerav, Verpeut, Jessica L., Ma, Jie, Jung, Caroline, Dunn, Connor E., Carvajal, Brigett V., Nguyen, Ai Vy, Boulanger, Lisa M. · 2024 · Molecular Therapy
39. Advances in AAV-mediated gene replacement therapy for pediatric monogenic neurological disorders
    Livia Zhou, Yafeng Wang, Yiran Xu, Yaodong Zhang, Changlian Zhu · 2024 · Molecular Therapy — Methods & Clinical Development
40. Nuclear pore dysfunction and disease: a complex opportunity
    Charlotte M. Fare, Jeffrey D. Rothstein · 2024 · Nucleus
41. Adeno-associated viral tools to trace neural development and connectivity across amphibians
    Eliza C.B. Jaeger, David Vijatovic, Astrid Deryckere, Nikol Zorin, Akemi L. Nguyen, Georgiy Ivanian, Jamie Woych, Rebecca C. Arnold, Alonso Ortega Gurrola, Arik Shvartsman et al. · 2024 · Developmental Cell
42. Attenuation of the Niemann-Pick type C2 disease phenotype by intracisternal administration of an AAVrh.10 vector expressing Npc2
    Sandra Markmann, Jasmine J. Christie-Reid, Jonathan B. Rosenberg, Bishnu P. De, Stephen M. Kaminsky, Ronald G. Crystal, Dolan Sondhi · 2018 · Experimental Neurology
43. DREADDs in Epilepsy Research: Network-Based Review
    John-Sebastian Mueller, Fabio Cesar Tescarollo, Hai Sun · 2022 · Frontiers in Molecular Neuroscience
44. Gene Therapy in the Management of Parkinson’s Disease: Potential of GDNF as a Promising Therapeutic Strategy
    Tapan Behl, Ishnoor Kaur, Arun Kumar, Vineet Mehta, Gökhan Zengin, Sandeep Arora · 2020 · Current Gene Therapy
45. Development of mitochondrial gene-editing strategies and their potential applications in mitochondrial hereditary diseases: a review
    Yanyan Gao, Linlin Guo, Fei Wang, Yin Wang, Peifeng Li, Dejiu Zhang · 2023 · Cytotherapy
46. Self-delivering, chemically modified CRISPR RNAs for AAV co-delivery and genome editing in vivo
    Han Zhang, Karen L. Kelly, Jonathan L. C. Lee, Dimas Echeverria, David Cooper, Rebecca Panwala, Nadia Amrani, Zexiang Chen, Nicholas Gaston, Atish A. Wagh et al. · 2023 · Nucleic Acids Research
47. Optogenetic recruitment of spinal reflex pathways from large‐diameter primary afferents in non‐transgenic rats transduced with AAV9/Channelrhodopsin 2
    Shinji Kubota, Wupuer Sidikejiang, Moeko Kudo, Ken‐ichi Inoue, Tatsuya Umeda, Masahiko Takada, Kazuhiko Seki · 2019 · The Journal of Physiology
48. New Indications for Hematopoietic Stem Cell Gene Therapy in Lysosomal Storage Disorders
    Linda Rossini, Caterina Durante, Antonio Marzollo, Alessandra Biffi · 2022 · Frontiers in Oncology
49. Design of a stable human acid‐β‐glucosidase: towards improved Gaucher disease therapy and mutation classification
    Šárka Pokorná, Olga Khersonsky, Rosalie Lipsh‐Sokolik, Adi Goldenzweig, Rebekka Nielsen, Yacov Ashani, Yoav Peleg, Tamar Unger, Shira Albeck, Orly Dym et al. · 2023 · FEBS Journal
50. Adeno-associated virus (AAV) reduces cortical dendritic complexity in a TLR9-dependent manner
    Christos Michael Suriano, Jessica L. Verpeut, Neerav Kumar, Jie Ma, Caroline Jung, Lisa M. Boulanger · 2021 · bioRxiv (Cold Spring Harbor Laboratory)
51. Capsid-Engineering for Central Nervous System-Directed Gene Therapy with Adeno-Associated Virus Vectors
    Josephine Macdonald, Jennifer Marx, Hildegard Büning · 2021 · Human Gene Therapy
52. Adeno-Associated Viruses for Modeling Neurological Diseases in Animals: Achievements and Prospects
    Evgenii A. Lunev, Anna A. Karan, Tatiana V. Egorova, Maryana V. Bardina · 2022 · Biomedicines
53. New AAV9 engineered variants with enhanced neurotropism and reduced liver off-targeting in mice and marmosets
    Serena Gea Giannelli, Mirko Luoni, Angelo Iannielli, Jinte Middeldorp, Ingrid H.C.H.M. Philippens, Simone Bido, Jakob Körbelin, Vania Broccoli · 2024 · iScience
54. Conditional RNA interference in mammalian cells via RNA transactivation
    Yu Zhou, Peike Sheng, Jiayi Li, Yudan Li, Mingyi Xie, Alexander A. Green · 2024 · Nature Communications
55. Direct Intrathecal Injection of Recombinant Adeno-associated Viruses in Adult Mice
    Dongxiao Li, Yundu Li, Yunyun Tian, Zuoshang Xu, Yansu Guo · 2019 · Journal of Visualized Experiments
56. Viral vectors as a novel tool for clinical and neuropsychiatric research applications
    Yao Wang, Zhiwei Hu, Peijun Ju, Shan Yin, Fujie Wang, Oudong Pan, Jinghong Chen · 2018 · General Psychiatry
57. Crucial aspects for maintaining rAAV stability
    Johannes Lengler, Mia Gavrila, Janina Brandis, Kristina Palavra, Felix Dieringer, Sabine Unterthurner, Felix F. Fuchsberger, Barbara Kraus, Juan A. Hernández Bort · 2024 · Scientific Reports
58. Gene Therapy for Neuronopathic Mucopolysaccharidoses: State of the Art
    M. D. de Castro, M. del Toro, R. Giugliani, M. Couce · 2021 · International Journal of Molecular Sciences
59. Survivin overexpression via adeno‐associated virus vector Rh10 ameliorates ischemic damage after middle cerebral artery occlusion in rats
    Yoshihide Sehara, Toshiki Inaba, Takao Urabe, Fumio Kurosaki, Masashi Urabe, Naoki Kaneko, Kuniko Shimazaki, Kensuke Kawai, Hiroaki Mizukami · 2018 · European Journal of Neuroscience
60. Targeting the gastrointestinal tract with viral vectors: state of the art and possible applications in research and therapy
    Roeland Buckinx, Jean‐Pierre Timmermans · 2016 · Histochemistry and Cell Biology
61. Small interfering RNAs based therapies for intracerebral hemorrhage: challenges and progress in drug delivery systems
    Zahoor Ahmad Shah, Daniyah A. Almarghalani, Sai H. S. Boddu, Mohammad Ali, Akhila Kondaka, Devin Ta, RayyanA Shah · 2021 · Neural Regeneration Research
62. Nanoparticle-based Gene Therapy for Neurodegenerative Disorders
    Nelofer Ereej, Huma Hameed, Mahtab Ahmad Khan, Saleha Faheem, Anam Hameed · 2024 · Mini-Reviews in Medicinal Chemistry
63. A Simple, Two-Step, Small-Scale Purification of Recombinant Adeno-Associated Viruses
    Shih‐Heng Chen, Amy B. Papaneri, Mitzie Walker, Erica Scappini, Robert D. Keys, Negin P. Martin · 2020 · Journal of Virological Methods
64. Intracerebral lentiviral ABCD1 gene therapy in an early disease onset ALD mouse model
    Jie Gong, Yunyun Liu, Tsai‐Hua Chung, Liu Xu, Troy C. Lund, Lung‐Ji Chang · 2022 · Gene Therapy
65. Gene Transfer in Rodent Nervous Tissue Following Hindlimb Intramuscular Delivery of Recombinant Adeno-Associated Virus Serotypes AAV2/6, AAV2/8, and AAV2/9
    Asad Jan, Mette Richner, Christian Bjerggaard Vægter, Jens Randel Nyengaard, Poul Henning Jensen · 2019 · Neuroscience Insights
66. Gene Therapy for Neuropsychiatric Disorders: Potential Targets and Tools
    Marzieh Lotfi, Murtaza M. Tambuwala, Seyed Hossein Shahcheraghi, Jamshid Ayatollahi, Alaa A. A. Aljabali, Mazhar Salim Al Zoubi, Pritam Kumar Panda, Vijay Kumar Mishra, Saurabh Satija, Nitin B. Charbe et al. · 2022 · CNS & Neurological Disorders - Drug Targets
67. Removal of empty capsids from high‐dose adeno‐associated virus 9 gene therapies
    William S. Kish, J Lightholder, Tamara Zeković, Alex Berrill, Matthew K. Roach, William B. Wellborn, Eric Vorst · 2024 · Biotechnology and Bioengineering
68. Identification of Cis-Regulatory Modules for Aav-Based Cell Type-Specific Targeting in the Retina and Brain
    Cheng‐Hui Lin, Yue Sun, Candace S. Y. Chan, Man-Ru Wu, Lei Gu, Alexander E. Davis, Baokun Gu, Wenlin Zhang, Bogdan Tanasă, Lei Ray Zhong et al. · 2021 · Journal of Biological Chemistry
69. A new protocol for whole-brain biodistribution analysis of AAVs by tissue clearing, light-sheet microscopy and semi-automated spatial quantification
    Miguel Monteiro Lopes, Jacques Paysan, José Rino, Sara Lopes, Luís Pereira de Almeida, Luísa Cortes, Rui Jorge Nobre · 2022 · Gene Therapy
70. Postnatal therapeutic approaches in genetic neurodevelopmental disorders
    Gilad Levy, Boaz Barak · 2020 · Neural Regeneration Research
71. Emerging Concepts in Vector Development for Glial Gene Therapy: Implications for Leukodystrophies
    Georg von Jonquières, Caroline D. Rae, Gary D. Housley · 2021 · Frontiers in Cellular Neuroscience
72. Premature delivery in the domestic sow in response to in utero delivery of AAV9 to fetal piglets
    Kelly A. Rich, Christopher G. Wier, Jessica R. Russo, Lingling Kong, Patrick L. Heilman, Anthony Reynolds, Amy Knapp, Megan G. Pino, Elizabeth Keckley, Lori Mattox et al. · 2021 · Gene Therapy
73. Taking the knife to neurodegeneration: a review of surgical gene therapy delivery to the CNS
    Andrea P. Perera, Olivier Brock, Aminul Islam Ahmed, Christopher E. Shaw, Keyoumars Ashkan · 2024 · Acta Neurochirurgica
74. Viral Vector Delivery of DREADDs for CNS Therapy
    Ceri A. Pickering, Nicholas D. Mazarakis · 2021 · Current Gene Therapy
75. Long-Term Disease Prevention with a Gene Therapy Targeting Oligodendrocytes in a Mouse Model of Adrenomyeloneuropathy
    Özgür-Günes, Yasemin, Chedik, Malha, Le Stunff, Catherine, Fovet, Claire-Maëlle, Bougnères, Pierre · 2022 · Human Gene Therapy
76. Evolving therapies in neuronopathic LSDs: opportunities and challenges
    Deepa Soundara Rajan, Maria Luisa Escolar · 2022 · Metabolic Brain Disease
77. Intrathecal delivery of a bicistronic AAV9 vector expressing β-hexosaminidase A corrects Sandhoff disease in a murine model: A dosage study
    Alex E. Ryckman, Natalie M. Deschenes, Brianna M. Quinville, Karlaina J.L. Osmon, Melissa Mitchell, Zhilin Chen, Steven James Gray, Jagdeep Singh Walia · 2023 · Molecular Therapy — Methods & Clinical Development
78. Tracking systems for intracranial medical devices: A review
    Quentin François, Arthur André, Bertrand Duplat, Sinan D Haliyo, Stéphane Régnier · 2019 · Medical Devices & Sensors
79. Single Systemic Administration of a Gene Therapy Leading to Disease Treatment in Metachromatic LeukodystrophyArsaKnock-Out Mice
    Thia St Martin, Tania A. Seabrook, Katherine Gall, Jenn Newman, Nancy Avila, April Hayes, Monicah Kivaa, Jason Lotterhand, Michael P. Mercaldi, Kruti Patel et al. · 2023 · Journal of Neuroscience
80. Dorsal Root Ganglion Injection and Dorsal Root Crush Injury as a Model for Sensory Axon Regeneration
    Menghon Cheah, James W. Fawcett, Melissa R. Andrews · 2017 · Journal of Visualized Experiments
81. RNA interference-based functional knockdown of the voltage-gated potassium channel Kv7.2 in dorsal root ganglion neurons after in vitro and in vivo gene transfer by adeno-associated virus vectors
    Markus Valdor, Anke Wagner, Viola Röhrs, Johanna Berg, Henry Fechner, Wolfgang Schröder, Thomas Tzschentke, Gregor Bahrenberg, Thomas Christoph, Jens Kurreck · 2017 · Molecular Pain
82. Synthetic dosage-compensating miRNA circuits for quantitative gene therapy
    Michael James Flynn, A. Mayfield, Rongrong Du, Viviana Gradinaru, Michael B. Elowitz · 2024 · bioRxiv (Cold Spring Harbor Laboratory)
83. Incomplete elimination of viral genomes is associated with chronic inflammation in nonhuman primate livers after AAV-mediated gene transfer
    Virginie Pichard, Mickaël Guilbaud, Marie Devaux, Nicolas Jaulin, Malo Journou, Magalie Michée‐Cospolite, Alexandra Garcia, Nicolas Ferry, Sophie Michalak-Provost, Gwladys Gernoux et al. · 2025 · Gene Therapy
84. Effective Viral Delivery of Genetic Constructs to Neuronal Culture for Modeling and Gene Therapy of GNAO1 Encephalopathy
    Evgenii A. Lunev, Anna A. Shmidt, Svetlana G. Vassilieva, Irina M. Savchenko, Vyacheslav A. Loginov, Valeria I. Marina, Tatiana V. Egorova, Maryana V. Bardina · 2022 · Molecular Biology
85. Helicid Reverses the Effect of Overexpressing NCALD, Which Blocks the sGC/cGMP/PKG Signaling Pathway in the CUMS-Induced Rat Model
    Xiao-Tong Zhang, Yuan Zhang, Yuanxiang Zhang, Zhen-Yi Jiang, Hui Yang, Lan Jiang, Bin Yang, Jiucui Tong · 2021 · Journal of Healthcare Engineering · Retracted
86. Considerations for the use of virally delivered genetic tools for in-vivo circuit analysis and behavior in mutant mice: a practical guide to optogenetics
    Dóra Zelena, Kornél Demeter, József Haller, Diána Balázsfi · 2017 · Behavioural Pharmacology
87. Blocking an epitope of misfolded SOD1 ameliorates disease phenotype in a model of amyotrophic lateral sclerosis
    Shamchal Bakavayev, Alexandra Stavsky, Shirel Argueti-Ostrovsky, Galit Yehezkel, Yael Fridmann‐Sirkis, Ze’ev Barak, Daniel Gitler, Adrian Israelson, Stanislav Engel · 2023 · Brain
88. A Novel Approach to the Treatment of Plasma Protein Deficiency: Ex Vivo-Manipulated Adipocytes for Sustained Secretion of Therapeutic Proteins
    Masayuki Kuroda, Yasushi Saitō, Masayuki Aso, Koutaro Yokote · 2018 · Chemical and Pharmaceutical Bulletin
89. Intracisternal IGF‐1 gene therapy abrogates kainic acid‐induced excitotoxic damage of the rat spinal cord
    Fabián Nishida, Carolina Natalia Zanuzzi, María Susana Sisti, Eugenia Falomir Lockhart, Agustina Elea Camiña, Claudia Beatriz Hereñú, María José Bellini, Enrique Leo Portiansky · 2020 · European Journal of Neuroscience
90. Advanced Nanobiomedical Approaches to Combat Coronavirus Disease of 2019
    Halle Lutz, Kristen D. Popowski, Phuong‐Uyen Dinh, Ke Cheng · 2020 · Advanced NanoBiomed Research
91. Selective targeting of striatal parvalbumin-expressing interneurons for transgene delivery
    Marcelo Duarte Azevedo, Sibilla Sander, Cheryl Jeanneret, Soophie Olfat, Liliane Tenenbaum · 2021 · Journal of Neuroscience Methods
92. Tropism, intracerebral distribution, and transduction efficiency of HIV- and SIV-based lentiviral vectors after injection into the mouse brain: a qualitative and quantitative in vivo study
    Juraj Hlavatý, Zbyněk Tonar, Matthias Renner, Sylvia Panitz, Helga Petznek, Matthias Schweizer, Silke Schüle, Björn‐Philipp Kloke, Rudolf Moldzio, Kirsti Witter · 2017 · Histochemistry and Cell Biology
93. AAV-PHP.eB achieves superior neuronal transduction over AAV9 in pigtail macaques following intracerebroventricular administration
    Michal G. Fortuna, Lyle H. Nyberg, Naz Taskin, Avery C. Hunker, Natalie Weed, Kathryn M. Gudsnuk, Melissa R. Berg, Boaz P. Levi, Ed S. Lein, Jonathan T. Ting · 2025 · Molecular Therapy — Methods & Clinical Development
94. Multi-targeting zinc finger nuclease vector unsilences paternal UBE3A in a mouse model of Angelman syndrome
    Hannah Bazick, Lucas M. James, Bonnie Taylor‐Blake, Justin M. Wolter, Mark J. Zylka · 2025 · Gene Therapy
95. Evaluation of AAV transduction efficiency via multiple delivery routes: Insights from peripheral and central nervous system analysis
    Na Kyung Lee, Duk Lyul Na, Jongwan Kim, Bumwhee Lee, Hee‐Jin Kim, Hyemin Jang, Jeehun Lee, Jong Wook Chang · 2025 · Neuroscience
96. Treatment of GM2 Gangliosidosis in Adult Sandhoff Mice Using an Intravenous Self-Complementary Hexosaminidase Vector
    Jagdeep Singh Walia, Karlaina J.L. Osmon, Patrick Thompson, Evan Woodley, Subha Karumuthil‐Melethil, Cliff Heindel, John G. Keimel, William F. Kaemmerer, Steven James Gray · 2021 · Current Gene Therapy
97. Reduced Immunogenicity of Intraparenchymal Delivery of Adeno-Associated Virus Serotype 2 Vectors: Brief Overview
    Shin‐ichi Muramatsu, Wuh‐Liang Hwu, Bruria Gidoni-Ben-Zeev · 2021 · Current Gene Therapy
98. Gene therapy of neurodegenerative diseases: achievements, developments, and clinical implementation challenges
    E. V. Melnikova, Вадим Анатольевич Меркулов, O. V. Merkulova · 2023 · Biological Products Prevention Diagnosis Treatment
99. Secreted therapeutics: monitoring durability of microRNA-based gene therapies in the central nervous system
    Marina Sogorb-González, Carlos Vendrell‐Tornero, Jolanda Snapper, Anouk Stam, Sonay Keskin, Jana Miniarikova, Elisabeth A. Spronck, Martin de Haan, Rienk Nieuwland, Pavlina S. Konstantinova et al. · 2021 · Brain Communications
100. Chemogenetic ligands for translational neurotheranostics
     Jordi Bonaventura, Mark A. G. Eldridge, Feng Hu, Juan L. Gomez, Marta Sánchez‐Soto, Ara M. Abramyan, Sherry Lam, Matthew A. Boehm, Christina M. Ruiz, Mitchell R. Farrell et al. · 2018 · bioRxiv (Cold Spring Harbor Laboratory)