未登录Cell-Based Therapy for Canavan Disease Using Human iPSC-Derived NPCs and OPCs使用人类iPSC衍生的npc和opc对Canavan病进行基于细胞的治疗
Feng, Lizhao; Chao, Jianfei; Tian, E.; Li, Li; Ye, Peng; Zhang, Mi; Chen, Xianwei; Cui, Qi; Sun, Guihua; Zhou, Tao; Felix, Gerardo; Qin, Yue; Li, Wendong; Meza, Edward David; Klein, Jeremy; Ghoda, Lucy; Hu, Weidong; Luo, Yonglun; Dang, Wei; Hsu, David; Gold, Joseph; Goldman, Steven A.; Matalon, Reuben; Shi, Yanhong .png)
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收藏Identification of novel candidate disease genes from de novo exonic copy number variants从新生外显子拷贝数变异中鉴定新的候选疾病基因Gambin, Tomasz; Yuan, Bo; Bi, Weimin; Liu, Pengfei; Rosenfeld, Jill A.; Coban-Akdemir, Zeynep; Pursley, Amber N.; Nagamani, Sandesh C. S.; Marom, Ronit; Golla, Sailaja; Dengle, Lauren; Petrie, Heather G.; Matalon, Reuben; Emrick, Lisa; Proud, Monica B.; Treadwell-Deering, Diane; Chao, Hsiao-Tuan; Koillinen, Hannele; Brown, Chester; Urraca, Nora; Mostafavi, Roya; Bernes, Saunder; Roeder, Elizabeth R.; Nugent, Kimberly M.; Bader, Patricia I.; Bellus, Gary; Cummings, Michael; Northrup, Hope; Ashfaq, Myla; Westman, Rachel; Wildin, Robert; Beck, Anita E.; Immken, LaDonna; Elton, Lindsay; Varghese, Shaun; Buchanan, Edward; Faivre, Laurence; Lefebvre, Mathilde; Schaaf, Christian P.; Walkiewicz, Magdalena; Yang, Yaping; Kang, Sung-Hae L.; Lalani, Seema R.; Bacino, Carlos A.; Beaudet, Arthur L.; Breman, Amy M.; Smith, Janice L.; Cheung, Sau Wai; Lupski, James R.; Patel, Ankita; Shaw, Chad A.; Stankiewicz, Pawel 分享 收藏 Redirecting N-acetylaspartate metabolism in the central nervous system normalizes myelination and rescues Canavan diseaseGessler, Dominic J.; Li, Danning; Xu, Hongxia; Su, Qin; Sanmiguel, Julio; Tuncer, Serafettin; Moore, Constance; King, Jean; Matalon, Reuben; Gao, Guangping 分享 收藏 Long-term treatment of phenylketonuria with a new medical food containing large neutral amino acidsConcolino, D.; Mascaro, I.; Moricca, M. T.; Bonapace, G.; Matalon, K.; Trapasso, J.; Radhakrishnan, G.; Ferrara, C.; Matalon, R.; Strisciuglio, P. 分享 收藏 rAAV Gene Therapy in a Canavan's Disease Mouse Model Reveals Immune Impairments and an Extended Pathology Beyond the Central Nervous SystemAhmed, Seemin Seher; Schattgen, Stefan A.; Frakes, Ashley E.; Sikoglu, Elif M.; Su, Qin; Li, Jia; Hampton, Thomas G.; Denninger, Andrew R.; Kirschner, Daniel A.; Kaspar, Brian; Matalon, Reuben; Gao, Guangping 分享 收藏 分享 收藏 分享 收藏 Acute Otitis Media and Other Complications of Viral Respiratory InfectionChonmaitree, Tasnee; Trujillo, Rocio; Jennings, Kristofer; Alvarez-Fernandez, Pedro; Patel, Janak A.; Loeffelholz, Michael J.; Nokso-Koivisto, Johanna; Matalon, Reuben; Pyles, Richard B.; Miller, Aaron L.; McCormick, David P. 分享 收藏 分享 收藏 Neurodevelopmental and neurobehavioral characteristics in males and females with CDKL5 duplicationsSzafranski, Przemyslaw; Golla, Sailaja; Jin, Weihong; Fang, Ping; Hixson, Patricia; Matalon, Reuben; Kinney, Daniel; Bock, Hans-georg; Craigen, William; Smith, Janice L.; Bi, Weimin; Patel, Ankita; Cheung, Sau Wai; Bacino, Carlos A.; Stankiewicz, Pawel 分享 收藏 Multiple Phenotypes in Phosphoglucomutase 1 Deficiency磷酸葡萄糖变现酶1缺乏症的多种表型Tegtmeyer, L. C.; Rust, S.; van Scherpenzeel, M.; Ng, B. G.; Losfeld, M. -E.; Timal, S.; Raymond, K.; He, P.; Ichikawa, M.; Veltman, J.; Huijben, K.; Shin, Y. S.; Sharma, V.; Adamowicz, M.; Lammens, M.; Reunert, J.; Witten, A.; Schrapers, E.; Matthijs, G.; Jaeken, J.; Rymen, D.; Stojkovic, T.; Laforet, P.; Petit, F.; Aumaitre, O.; Czarnowska, E.; Piraud, M.; Podskarbi, T.; Stanley, C. A.; Matalon, R.; Burda, P.; Seyyedi, S.; Debus, V.; Socha, P.; Sykut-Cegielska, J.; van Spronsen, F.; de Meirleir, L.; Vajro, P.; DeClue, T.; Ficicioglu, C.; Wada, Y.; Wevers, R. A.; Vanderschaeghe, D.; Callewaert, N.; Fingerhut, R.; van Schaftingen, E.; Freeze, H. H.; Morava, E.; Lefeber, D. J.; Marquardt, T. 分享 收藏 A Single Intravenous rAAV Injection as Late as P20 Achieves Efficacious and Sustained CNS Gene Therapy in Canavan MiceAhmed, Seemin Seher; Li, Huapeng; Cao, Chunyan; Sikoglu, Elif M.; Denninger, Andrew R.; Su, Qin; Eaton, Samuel; Navarro, Ana A. Liso; Xie, Jun; Szucs, Sylvia; Zhang, Hongwei; Moore, Constance; Kirschner, Daniel A.; Seyfried, Thomas N.; Flotte, Terence R.; Matalon, Reuben; Gao, Guangping 分享 收藏 分享 收藏 分享 收藏 分享 收藏 分享 收藏 分享 收藏 分享 收藏 分享 收藏 分享 收藏 
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