Selection of an Efficient AAV Vector for Robust CNS Transgene Expression
Hanlon, Killian S., Meltzer, Jonah C., Buzhdygan, Tetyana, Cheng, Ming J., Sena-Esteves, Miguel, Bennett, Rachel E., Sullivan, Timothy P., Razmpour, Roshanak, Gong, Yi, Ng, Carrie, Nammour, Josette, Maiz, Daniela, Dujardin, Simon, Ramirez, Servio H., Hudry, Eloise, Maguire, Casey A.·2019·Molecular Therapy - Methods & Clinical Development
Cited by (148)
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- The Roads We Take: Cellular Targets and Pathways Leading Biologics Across the Blood–Brain BarrierHabib Baghirov · 2022 · Frontiers in Drug Delivery
- Directed Evolution of AAV9 for Efficient Gene Expression in Cardiomyocytes In Vitro and In VivoLeonard Hüttermann, Lena C. Schröder, Prithviraj Manohar Vijaya Shetty, Timo Jonker, Susanne S. Hille, Anca Kliesow Remes, Andrea Matzen, Arnie Boender, Dirk Grimm, Derk Frank et al. · 2025 · Human Gene Therapy
- Considerations When Developing Blood–Brain Barrier Crossing Drug Delivery TechnologyRobert D. Bell · 2021 · Handbook of experimental pharmacology
- Isolation of Next-Generation Gene Therapy Vectors through Engineering, Barcoding, and Screening of Adeno-Associated Virus (AAV) Capsid VariantsKleopatra Rapti, Olena Maiakovska, Jonas Becker, Joanna Szumska, Margarita Zayas, Felix Bubeck, Jixin Liu, Emma Gerstmann, Chiara Krämer, Ellen Wiedtke et al. · 2022 · Journal of Visualized Experiments
- In vivo selection in non-human primates identifies superior AAV capsids for on-target CSF delivery to spinal cordKillian S. Hanlon, Ming Liang Cheng, Demitri de la Cruz, Nikita J. Patel, Miguel C. Santoscoy, Yi Gong, Carrie Ng, Diane M. Nguyen, Josette Nammour, Sean W. Clark et al. · 2023 · bioRxiv (Cold Spring Harbor Laboratory)
- Expression-based selection identifies a microglia-tropic AAV capsid for direct and CSF routes of administration in miceMiguel C. Santoscoy, Paula Espinoza, Killian S. Hanlon, Luna Yang, Lisa Nieland, Carrie Ng, Christian E. Badr, Suzanne E. Hickman, Demitri de la Cruz, Ana Griciuc et al. · 2024 · bioRxiv (Cold Spring Harbor Laboratory)
- Deep-sequencing-guided library screening and profiling of AAV capsids in the primate retinaYoussef Fouani, Dragica Blazevic, Martin Oti, Robert Becker, Benjamin Strobel, Tanja Schönberger, Marius Stephan, Sven Wichert, Felix F. Reichel, Immanuel Philipp Seitz et al. · 2026 · Molecular Therapy Advances
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- Validation studies and multi-omics analysis of Zhx2 as a candidate quantitative trait gene underlying brain oxycodone metabolite (oxymorphone) levels and behaviorWilliam B. Lynch, Sophia A. Miracle, Stanley I. Goldstein, Jacob A. Beierle, Rhea Bhandari, Ethan Gerhardt, Ava D. Farnan, Binh-Minh Nguyen, Kelly K. Wingfield, Ida Kazerani et al. · 2024 · Journal of Pharmacology and Experimental Therapeutics
- Next generation AAV-F capsid gene therapy rescues disease pathology in a model of pyruvate dehydrogenase complex deficiencyAnna Keegan, Özge Çetin, Ellie M. Chilcott, Juan F. Antinao Diaz, Simon D. Eaton, Simon N. Waddington, John Robert Counsell, Shamima Rahman, Rajvinder Karda · 2026 · Molecular Therapy Advances
- Directed evolution of AAV capsids for improved efficacy and specificity of delivery to preclinical models of human liverMagdalena Kwiatek, Aleksander Michalski, Karolina Abramowicz, Paweł Rutyna, Magdalena Wasiak, M. Reichert, Leszek Lisowski · 2023 · Lekarz Wojskowy
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- Intratumoral gene delivery of 4-1BBL boosts IL-12-triggered anti-glioblastoma immunityTaral R. Lunavat, Lisa Nieland, Sanne M. van de Looij, Alexandra J E M de Reus, Charles Couturier, Chadi A. El Farran, Tyler E. Miller, Julia K. Lill, Maryam Schübel, Tianhe Xiao et al. · 2025 · bioRxiv (Cold Spring Harbor Laboratory)
- Intra-arterial Gene Delivery for Neurodegenerative DiseasesJonathan M. Fischell · 2025 · Springer eBooks
- AAV gene therapy for Cockayne syndromeAna Rita Batista, Aine C. Scholand, William S. Callahan, McKenna K. Watson, Cassandra M. Sion, Tyler Mola, Kennedy O’Hara, Oliver D. King, Robert M King, Miguel Sena‐Esteves · 2025 · bioRxiv (Cold Spring Harbor Laboratory)
- rAAV-Mediated Gene Therapy Targeting CNS DisordersLester Suárez-Amarán, Christina A. Chatzi, Inge Flier, Richard Jude Samulski · 2025 · Springer eBooks
- EvoPRAISE: computationally guided directed evolution for rational AAV capsid engineeringHiroaki Ono, Shoko Fujino, Genshiro A. Sunagawa · 2025 · bioRxiv (Cold Spring Harbor Laboratory)
- VCAP-102 Achieves Exceptional BBB Penetration in Marmosets After Ten Years of AAV Capsid EvaluationYasunori Matsuzaki, Ayumu Konno, Yasuo Uchida, Tetsuya Terasaki, Hirokazu Hirai · 2025 · bioRxiv (Cold Spring Harbor Laboratory)
- Spatial mapping of barcoded, brain-tropic AAVs using multiplexed RNA in situ hybridizationYoussef Fouani, Martin Oti, Sebastian Jarosch, Dragica Blazevic, Matthias J. Duechs, Stephanie Ketterer, Robert Becker, Sarah Abele, Benjamin Strobel, Tanja Schoenberger et al. · 2026 · Molecular Therapy Advances
- Astrocytic and endothelial GLUT1 restoration improves brain glucose homeostasis in GLUT1 deficiency syndromeShoko Tamura, Hiroko Shimbo, N Aruga, Mai Kawaguchi, Haruo Okado, Yuga Yasuda, Yuichiro Miyaoka, Ayano Sasakura, Kinya Matsuo, Hideaki Nishihara et al. · 2026 · bioRxiv (Cold Spring Harbor Laboratory)
- Computationally guided discovery of Ly6e/LY6E-dependent AAV capsid variantsHiroaki Ono, Shoko Fujino, Genshiro A. Sunagawa · 2026 · iScience
- Could the overexpression of neuroglobin, via an intravenous administration of an AAV2/9 vector, durably attenuate cerebellar ataxia in Harlequin mice by preserving mitochondrial homeostasis?Edward Ratcliffe · 2025 · theses.fr (ABES)
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- Selecting Novel Retino-tropic AAV Capsids by Directed EvolutionAdrian Westhaus, Leszek Lisowski · 2026 · Methods in molecular biology
- Systemic AAV8-SIL1-TAT therapy improves motor function in a mouse model of Marinesco-Sjögren syndromeFabio Bellia, Anna Giulia Ruggieri, Laura Amodei, Francesca Potenza, Beatrice Dufrusine, Valeria Panella, Francesco Pizzo, Alessia Lamolinara, Manuela Iezzi, Luca Federici et al. · 2026 · Biochemical Pharmacology
- Trends in the Engineering of Adeno-Associated Virus (AAV) for Precision Gene Delivery to the Central Nervous System (CNS)Ezekiel S. Oloruntimehin, Alexander S. Malogolovkin · 2026 · International Journal of Molecular Sciences
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