未登录 分享 收藏
分享 收藏
MeCP2 gene therapy ameliorates disease phenotype in mouse model for Pitt Hopkins syndrome Dennys, Cassandra N.; Vermudez, Sheryl Anne D.; Deacon, Robert J. M.; Sierra-Delgado, J. Andrea; Rich, Kelly; Zhang, Xiaojin; Buch, Aditi; Weiss, Kelly; Moxley, Yuta; Rajpal, Hemangi; Espinoza, Francisca D.; Powers, Samantha; Avila, Ariel S.; Gogliotti, Rocco G.; Cogram, Patricia; Niswender, Colleen M.; Meyer, Kathrin C. 分享 收藏
C9ORF72 patient-derived endothelial cells drive blood-brain barrier disruption and contribute to neurotoxicity Aragon-Gonzalez, Ana; Shaw, Allan C.; Kok, Jannigje R.; Roussel, Florence S.; Souza, Cleide dos Santos; Granger, Sarah M.; Vetter, Tatyana; de Diego, Yolanda; Meyer, Kathrin C.; Beal, Selina N.; Shaw, Pamela J.; Ferraiuolo, Laura 分享 收藏
RNA helicase IGHMBP2 regulates THO complex to ensure cellular mRNA homeostasis Prusty, Archana Bairavasundaram; Hirmer, Anja; Sierra-Delgado, Julieth Andrea; Huber, Hannes; Guenther, Ulf-Peter; Schlosser, Andreas; Dybkov, Olexandr; Yildirim, Ezgi; Urlaub, Henning; Meyer, Kathrin C.; Jablonka, Sibylle; Erhard, Florian; Fischer, Utz 分享 收藏
Novel MECP2 gene therapy is effective in a multicenter study using two mouse models of Rett syndrome and is safe in non-human primates Powers, Samantha; Likhite, Shibi; Gadalla, Kamal K.; Miranda, Carlos J.; Huffenberger, Amy J.; Dennys, Cassandra; Foust, Kevin D.; Morales, Pablo; Pierson, Christopher R.; Rinaldi, Federica; Perry, Stephanie; Bolon, Brad; Wein, Nicolas; Cobb, Stuart; Kaspar, Brian K.; Meyer, Kathrin C. 分享 收藏
分享 收藏
分享 收藏
Mechanisms of IRF2BPL-related disorders and identification of a potential therapeutic strategy Ray, Shrestha Sinha; Dutta, Debdeep; Dennys, Cassandra; Powers, Samantha; Roussel, Florence; Lisowski, Pawel; Glazar, Petar; Zhang, Xiaojin; Biswas, Pipasha; Caporale, Joseph R.; Rajewsky, Nikolaus; Bickle, Marc; Wein, Nicolas; Bellen, Hugo J.; Likhite, Shibi; Marcogliese, Paul C.; Meyer, Kathrin C. 分享 收藏
分享 收藏
分享 收藏
CuATSM effectively ameliorates ALS patient astrocyte-mediated motor neuron toxicity in human in vitro models of amyotrophic lateral sclerosis Dennys, Cassandra N.; Roussel, Florence; Rodrigo, Rochelle; Zhang, Xiaojin; Delgado, Andrea Sierra; Hartlaub, Annalisa; Saelim-Ector, Asya; Ray, Will; Heintzman, Sarah; Fox, Ashley; Kolb, Stephen J.; Beckman, Joseph; Franco, Maria Clara; Meyer, Kathrin 分享 收藏
PolyGA targets the ER stress-adaptive response by impairing GRP75 function at the MAM in C9ORF72-ALS/FTD Pilotto, Federica; Schmitz, Alexander; Maharjan, Niran; Diab, Rim; Odriozola, Adolfo; Tripathi, Priyanka; Yamoah, Alfred; Scheidegger, Olivier; Oestmann, Angelina; Dennys, Cassandra N.; Ray, Shrestha Sinha; Rodrigo, Rochelle; Kolb, Stephen; Aronica, Eleonora; Di Santo, Stefano; Widmer, Hans Rudolf; Charlet-Berguerand, Nicolas; Selvaraj, Bhuvaneish T.; Chandran, Siddharthan; Meyer, Kathrin; Zuber, Benoit; Goswami, Anand; Weis, Joachim; Saxena, Smita 分享 收藏
AAV9-MCT8 Delivery at Juvenile Stage Ameliorates Neurological and Behavioral Deficits in a Mouse Model of MCT8-Deficiency Liao, Xiao-Hui; Avalos, Pablo; Shelest, Oksana; Ofan, Raz; Shilo, Michael; Bresee, Catherine; Likhite, Shibi; Vit, Jean-Philippe; Heuer, Heike; Kaspar, Brian; Meyer, Kathrin; Dumitrescu, Alexandra M.; Refetoff, Samuel; Svendsen, Clive N.; Vatine, Gad D. 分享 收藏
Optimization and validation of CAR transduction into human primary NK cells using CRISPR and AAV Kararoudi, Meisam Naeimi; Likhite, Shibi; Elmas, Ezgi; Yamamoto, Kenta; Schwartz, Maura; Sorathia, Kinnari; Pereira, Marcelo de Souza Fernandes; Sezgin, Yasemin; Devine, Raymond D.; Lyberger, Justin M.; Behbehani, Gregory K.; Chakravarti, Nitin; Moriarity, Branden S.; Meyer, Kathrin; Lee, Dean A. 分享 收藏
Neurotoxic Astrocytes Directly Converted from Sporadic and Familial ALS Patient Fibroblasts Reveal Signature Diversities and miR-146a Theragnostic Potential in Specific Subtypes 从散发性和家族性ALS患者成纤维细胞直接转化的神经毒性星形胶质细胞在特定亚型中显示出特征多样性和miR-146a的治疗潜力 Gomes, Catia; Sequeira, Catarina; Likhite, Shibi; Dennys, Cassandra N.; Kolb, Stephen J.; Shaw, Pamela J.; Vaz, Ana R.; Kaspar, Brian K.; Meyer, Kathrin; Brites, Dora 分享 收藏
Loss of IRF2BPL impairs neuronal maintenance through excess Wnt signaling Marcogliese, Paul C.; Dutta, Debdeep; Ray, Shrestha Sinha; Dang, Nghi D. P.; Zuo, Zhongyuan; Wang, Yuchun; Lu, Di; Fazal, Fatima; Ravenscroft, Thomas A.; Chung, Hyunglok; Kanca, Oguz; Wan, JiJun; Douine, Emilie D.; Network, Undiagnosed Diseases; Pena, Loren D. M.; Yamamoto, Shinya; Nelson, Stanley F.; Might, Matthew; Meyer, Kathrin C.; Yeo, Nan Cher; Bellen, Hugo J. 分享 收藏
分享 收藏
Follistatin-induced muscle hypertrophy in aged mice improves neuromuscular junction innervation and function Iyer, Chitra C.; Chugh, Deepti; Bobbili, Prameela J.; Blatnik, Anton J., III; Crum, Alexander E.; Yi, Allen F.; Kaspar, Brian K.; Meyer, Kathrin C.; Burghes, Arthur H. M.; Arnold, W. David 分享 收藏
分享 收藏
.png){kind=icon}
未登录.png){kind=icon}
.png){kind=icon}
.png){kind=icon}
.png){kind=icon}
.png){kind=icon}
