AAV-based gene therapy ameliorated CNS-specific GPI defect in mouse models by Yoshiko Murakami, Saori Umeshita, Kae Imanishi, Yoshichika Yoshioka, Akinori Ninomiya, Takehiko Sunabori, Shibi Likhite, Masato Koike, Kathrin C. Meyer, Taroh Kinoshita

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The postnatal injection of AAV9-FOXG1 rescues corpus callosum agenesis and other brain deficits in the mouse model of FOXG1 syndrome by Shin Jeon, Jaein Park, Shibi Likhite, Ji Hwan Moon, Dongjun Shin, Liwen Li, Kathrin C. Meyer, Jae W. Lee, Soo-Kyung Lee

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In-depth comparison of Anc80L65 and AAV9 retinal targeting and characterization of cross-reactivity to multiple AAV serotypes in humans by Maura K. Schwartz, Shibi Likhite, Tatyana A. Vetter, Megan C. Baird, Vicki McGovern, Andrea Sierra Delgado, Tom Mendel, Arthur Burghes, Kathrin C. Meyer

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RNA helicase IGHMBP2 regulates THO complex to ensure cellular mRNA homeostasis by Archana Bairavasundaram Prusty, Anja Hirmer, Julieth Andrea Sierra-Delgado, Hannes Huber, Ulf-Peter Guenther, Andreas Schlosser, Olexandr Dybkov, Ezgi Yildirim, Henning Urlaub, Kathrin C. Meyer, Sibylle Jablonka, Florian Erhard, Utz Fischer

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Combination AAV therapy with galectin-1 and SOD1 downregulation demonstrates superior therapeutic effect in a severe ALS mouse model by Megan C. Baird, Shibi B. Likhite, Tatyana A. Vetter, Joseph R. Caporale, Holly B. Girard, Florence S. Roussel, Abigail E. Howard, Maura K. Schwartz, Addison R. Reed, Abuzar Kaleem, Xiaojin Zhang, Kathrin C. Meyer

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C9ORF72 patient-derived endothelial cells drive blood-brain barrier disruption and contribute to neurotoxicity by Ana Aragón-González, Allan C Shaw, Jannigje R Kok, Florence S Roussel, Cleide dos Santos Souza, Sarah M Granger, Tatyana Vetter, Yolanda de Diego, Kathrin C Meyer, Selina N Beal, Pamela J Shaw, Laura Ferraiuolo

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