未登录 分享 收藏
Adeno-associated vector corneal gene therapy reverses corneal clouding in a feline model of mucopolysaccharidosis VI 腺相关病毒载体角膜基因疗法逆转了黏多糖贮积症VI型猫模型的角膜混浊。 Gilger, Brian C.; Song, Liujiang; Hasegawa, Tomoko; Salmon, Jacklyn H.; Bower, Jacquelyn J.; Vridhachalam, Naveen; Collins, Elisabeth; Roberts, Darby; Bagel, Jessica; Molony, Caitlyn; Vite, Charles; Hirsch, Matthew L. 分享 收藏
分享 收藏
Accumulation of alkyl-lysophosphatidylcholines in Niemann-Pick disease type C1 Mishra, Sonali; Kell, Pamela; Scherrer, David; Dietzen, Dennis J.; Vite, Charles H.; Berry-Kravis, Elizabeth; Davidson, Cristin; Cologna, Stephanie M.; Porter, Forbes D.; Ory, Daniel S.; Jiang, Xuntian 分享 收藏
Combination HSCT and intravenous AAV-mediated gene therapy in a canine model proves pivotal for translation of Krabbe disease therapy Bradbury, Allison M.; Bagel, Jessica; Swain, Gary; Miyadera, Keiko; Pesayco, Jill P.; Assenmacher, Charles-Antoine; Brisson, Becky; Hendricks, Ian; Wang, Xiao H.; Herbst, Zachary; Pyne, Nettie; Odonnell, Patricia; Shelton, Diane; Gelb, Michael; Hackett, Neil; Szabolcs, Paul; Vite, Charles H.; Escolar, Maria 分享 收藏
分享 收藏
Meningeal B Cell Clusters Correlate with Submeningeal Pathology in a Natural Model of Multiple Sclerosis Church, Molly E.; Ceja, Guadalupe; McGeehan, Megan; Miller, Miles C.; Farias, Priscilla; Sanchez, Melissa D.; Swain, Gary P.; Assenmacher, Charles-Antoine; Stopa, Edward G.; Vite, Charles H.; Bar-Or, Amit; Alvarez, Jorge, I 分享 收藏
Krabbe disease successfully treated via monotherapy of intrathecal gene therapy Bradbury, Allison M.; Bagel, Jessica H.; Duc Nguyen; Lykken, Erik A.; Salvador, Jill Pesayco; Jiang, Xuntian; Swain, Gary P.; Assenmacher, Charles A.; Hendricks, Ian J.; Miyadera, Keiko; Hess, Rebecka S.; Ostrager, Arielle; ODonnell, Patricia; Sands, Mark S.; Ory, Daniel S.; Shelton, G. Diane; Bongarzone, Ernesto R.; Gray, Steven J.; Vite, Charles H. 分享 收藏
Global CNS correction in a large brain model of human alpha-mannosidosis by intravascular gene therapy Yoon, Sea Young; Hunter, Jacqueline E.; Chawla, Sanjeev; Clarke, Dana L.; Molony, Caitlyn; O'Donnell, Patricia A.; Bagel, Jessica H.; Kumar, Manoj; Poptani, Harish; Vite, Charles H.; Wolfe, John H. 分享 收藏
Application of N-palmitoyl-O-phosphocholineserine for diagnosis and assessment of response to treatment in Niemann-Pick type C disease Sidhu, Rohini; Kell, Pamela; Dietzen, Dennis J.; Farhat, Nicole Y.; An Ngoc Dang Do; Porter, Forbes D.; Berry-Kravis, Elizabeth; Vite, Charles H.; Reunert, Janine; Marquardt, Thorsten; Giugliani, Roberto; Lourenco, Charles M.; Bodamer, Olaf; Wang, Raymond Y.; Plummer, Ellen; Schaffer, Jean E.; Ory, Daniel S.; Jiang, Xuntian 分享 收藏
分享 收藏
N-acyl-O-phosphocholineserines: structures of a novel class of lipids that are biomarkers for Niemann-Pick C1 disease Sidhu, Rohini; Mondjinou, Yawo; Qian, Mingxing; Song, Haowei; Kumar, Arun Babu; Hong, Xinying; Hsu, Fong-Fu; Dietzen, Dennis J.; Yanjanin, Nicole M.; Porter, Forbes D.; Berry-Kravis, Elizabeth; Vite, Charles H.; Gelb, Michael H.; Schaffer, Jean E.; Ory, Daniel S.; Jiang, Xuntian 分享 收藏
A cross-species approach to disorders affecting brain and behaviour Devinsky, Orrin; Boesch, Jordyn M.; Cerda-Gonzalez, Sofia; Coffey, Barbara; Davis, Kathryn; Friedman, Daniel; Hainline, Brian; Houpt, Katherine; Lieberman, Daniel; Perry, Pamela; Pruess, Harald; Samuels, Martin A.; Small, Gary W.; Volk, Holger; Summerfield, Artur; Vite, Charles; Wisniewski, Thomas; Natterson-Horowitz, Barbara 分享 收藏
Neuroimaging and neuropathology reveal progressively abnormal white matter and cerebrospinal fluid volume in MPS I dogs Dickson, Patricia; Chen, Steven; Nestrasil, Igor; Le, Steven; Kan, Shih-hsin; Jens, Jackie; Snella, Elizabeth; Ellinwood, N. Matthew; Guzman, Miguel; White, Leonard; Cooper, Jonathan D.; Egeland, Martin; Vite, Charles; Provenzale, James 分享 收藏
分享 收藏
Intrathecal 2-hydroxypropyl-β-cyclodextrin decreases neurological disease progression in Niemann-Pick disease, type C1: a non-randomised, open-label, phase 1-2 trial Ory, Daniel S.; Ottinger, Elizabeth A.; Farhat, Nicole Yanjanin; King, Kelly A.; Jiang, Xuntian; Weissfeld, Lisa; Berry-Kravis, Elizabeth; Davidson, Cristin D.; Bianconi, Simona; Keener, Lee Ann; Rao, Ravichandran; Soldatos, Ariane; Sidhu, Rohini; Walters, Kimberly A.; Xu, Xin; Thurm, Audrey; Solomon, Beth; Pavan, William J.; Machielse, Bernardus N.; Kao, Mark; Silber, Steven A.; Mckew, John C.; Brewer, Carmen C.; Vite, Charles H.; Walkley, Steven U.; Austin, Christopher P.; Porter, Forbes D. 分享 收藏
分享 收藏
Temporal behavior of seizures and interictal bursts in prolonged intracranial recordings from epileptic canines 癫痫犬的长时间颅内记录中癫痫发作和发作间爆发的时间行为 Ung, Hoameng; Davis, Kathryn A.; Wulsin, Drausin; Wagenaar, Joost; Fox, Emily; McDonnell, John J.; Patterson, Ned; Vite, Charles H.; Worrell, Gregory; Litt, Brian 分享 收藏
Cerebrospinal Fluid Calbindin D Concentration as a Biomarker of Cerebellar Disease Progression in Niemann-Pick Type C1 Disease Bradbury, Allison; Bagel, Jessica; Sampson, Maureen; Farhat, Nicole; Ding, Wenge; Swain, Gary; Prociuk, Maria; O'Donnell, Patricia; Drobatz, Kenneth; Gurda, Brittney; Wassif, Christopher; Remaley, Alan; Porter, Forbes; Vite, Charles 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}
