mTOR downregulation promotes anti-inflammatory responses via the CCL3-CCR5 axis in hypoxic retinopathy by Tae Kwon Moon, Im Kyeung Kang, Kyoung Jin Lee, Ji Hyun Kim, Hee Jong Kim, A. Reum Han, Ha-Na Woo, Joo Yong Lee, Jun-Sub Choi, Keerang Park, Heuiran Lee

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Probing aspects of extracellular vesicle associated AAV allows increased vector yield and insight into its transduction and immune-evasive properties by Ming Cheng, Demitri de la Cruz, Adam V. Crain, Paula Espinoza, Carrie Ng, Zachary C. Elmore, Aravind Asokan, Casey A. Maguire

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A novel platform for engineered AAV-based vaccines by Sabrina Babutzka, Miranda Gehrke, Anastasia Papadopoulou, Maria Diedrichs-Möhring, Maria Giannaki, Lena Hennis, Bastian Föhr, Cale Kooyman, Andreas Osterman, Evangelia Yannaki, Gerhild Wildner, Hermann Ammer, Stylianos Michalakis

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Reducing off-target expression of mRNA therapeutics and vaccines in the liver with microRNA binding sites by Brian J. Parrett, Satoko Yamaoka, Michael A. Barry

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Modeling Pkd1 gene-targeted strategies for correction of polycystic kidney disease by Almira Kurbegovic, Rey Christian Pacis, Marie Trudel

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Development of cell lines with increased susceptibility to diverse adeno-associated viral vectors to enable in vitro potency assays by James Zengel, Emma S. Esterman, Anitha Ponnuswami, Nicholas R. Wall, Jan E. Carette

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Characterization and effective expansion of CD4−CD8− TCRαβ+ T cells from individuals living with type 1 diabetes by J. Ernesto Fajardo-Despaigne, Félix Lombard-Vadnais, Adam-Nicolas Pelletier, Aïnhoa Olazabal, Lucie Boutin, Sarah Pasquin, Valérie Janelle, Laurent Legault, Jean-Sébastien Delisle, Erin E. Hillhouse, Lise Coderre, Sylvie Lesage

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A versatile cell line for establishing potency of cell type-specific AAV transgenes by Refugio A. Martinez, Yemeserach M. Bishaw, Meagan A. Quinlan, Erin L. Groce, Bryan B. Gore, Ed S. Lein, John K. Mich, Boaz P. Levi

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Capivasertib enhances chimeric antigen receptor T cell activity in preclinical models of B cell lymphoma by Hui-Ju Hsieh, Ryan Urak, Mary C. Clark, Larry W. Kwak, Stephen J. Forman, Xiuli Wang

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Intravenous esketamine in pediatric Rett syndrome: An open-label, early phase 1 pilot study by Huiping Li, Shu Liu, Caimei Lin, Yuchao Wu, Xiuping Wu, Yukun Huang, Yajun Wu, Xiubin Tong, Xiu Xu

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Silencing hepatic PCSK9 via novel chimeric AAV8 mitigates the progression of atherosclerosis by inhibiting inflammation in ApoE−/− mice by Xiaocui Chen, Minghui Sun, Xiang Ma, Yitong Ma, Bangdang Chen

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Ensuring patient access to gene therapies for rare diseases: Navigating reimbursement and coverage challenges by Diane Berry, Carolyn Hickey, Lisa Kahlman, James Long, Christina Markus, Caitlin K. McCombs

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Human striatal progenitor cells that contain inducible safeguards and overexpress BDNF rescue Huntington’s disease phenotypes by Danielle A. Simmons, Sridhar Selvaraj, Tingshuo Chen, Gloria Cao, Talita Souto Camelo, Tyne L.M. McHugh, Selena Gonzalez, Renata M. Martin, Juste Simanauskaite, Nobuko Uchida, Matthew H. Porteus, Frank M. Longo

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CRISPR screen reveals modifiers of rAAV production including known rAAV infection genes playing an unexpected role in vector production by Emily E. O’Driscoll, Sakshi Arora, Jonathan F. Lang, Beverly L. Davidson, Ophir Shalem

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Systemic delivery of AAV5, AAV8, and AAV9 packaging a C5-12-microdystrophin-FLAG expression cassette in non-human primates by Mengping Liu, Erica Cook, Yanshan Dai, Erich Ehlert, Francois du Plessis, Jacek Lubelski, Bogdan G. Sleczka, Petia Shipkova, Zhuyin Li, Joshua Gamse, David Gordon, Leonard P. Adam, Paul C. Levesque, Glen B. Banks

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The ambition for a one-and-done vision-saving AAV vector by Therese Cronin

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The combination of rAAV pseudo-lipid nanoparticle and triamcinolone acetonide enables multi-administration to liver by Chunmei Gan, Mi Leng, Yu Liu, Zhaoyue Zheng, Siwu He, Wen Qiao, Lin Xiao, Yao Xiao, Jingya Ye, Lixing Zhou, Jiao Zhou, Boduan Xiao, Wenxin Zhao, Jiamei Yang, Aohan Wu, Huiyuan Zhang, Hongbo Hu, Xiaobo Cen, Zhiyong Qian, Haohao Dong, C. Alexander Valencia, Lunzhi Dai, Hoi Yee Chow, Lei Zhang, Biao Dong

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Recombinant AAV batch profiling by nanopore sequencing elucidates product-related DNA impurities and vector genome length distribution by Florian Dunker-Seidler, Kathrin Breunig, Magdalena Haubner, Florian Sonntag, Markus Hörer, Rebecca C. Feiner

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