日期:
2020 年 — 2026 年
2020
2021
2022
2023
2024
2025
2026
影响因子:

Effect of disease progression on CSF-directed AAV gene therapy in a large brain animal model of lysosomal storage disease.

在溶酶体贮积症大型脑动物模型中,疾病进展对脑脊液靶向AAV基因治疗的影响

Hunter Jacqueline E, Molony Caitlyn M, Panek Wojciech K, Vite Charles H, Chawla Sanjeev, Poptani Harish, Wolfe John H

Intracisternal vs intraventricular injection of AAV1 result in comparable, widespread transduction of the dog brain

脑池内注射与脑室内注射AAV1可导致犬脑内相似的广泛转导。

Hunter, Jacqueline E; Vite, Charles H; Molony, Caitlyn M; O'Donnell, Patricia A; Wolfe, John H

Beta-mannosidosis in a domestic cat associated with a missense variant in MANBA

家猫β-甘露糖苷酶缺乏症与MANBA基因错义变异相关

Katz, Martin L; Cook, James; Vite, Charles H; Campbell, Rebecca S; Coghill, Lyndon M; Lyons, Leslie A

Low incidence of hepatocellular carcinoma in mice and cats treated with systemic adeno-associated viral vectors.

用全身性腺相关病毒载体治疗的小鼠和猫中肝细胞癌的发生率较低

Ferla Rita, Alliegro Marialuisa, Dell'Anno Margherita, Nusco Edoardo, Cullen John M, Smith Stephanie N, Wolfsberg Tyra G, O'Donnell Patricia, Wang Ping, Nguyen Anh-Dao, Chandler Randy J, Chen Zelin, Burgess Shawn M, Vite Charles H, Haskins Mark E, Venditti Charles P, Auricchio Alberto

Application of N-palmitoyl-O-phosphocholineserine for diagnosis and assessment of response to treatment in Niemann-Pick type C disease

N-棕榈酰-O-磷酸胆碱丝氨酸在尼曼-匹克C型疾病的诊断和治疗反应评估中的应用

Sidhu, Rohini; Kell, Pamela; Dietzen, Dennis J; Farhat, Nicole Y; Do, An Ngoc Dang; Porter, Forbes D; Berry-Kravis, Elizabeth; Vite, Charles H; Reunert, Janine; Marquardt, Thorsten; Giugliani, Roberto; Lourenço, Charles M; Bodamer, Olaf; Wang, Raymond Y; Plummer, Ellen; Schaffer, Jean E; Ory, Daniel S; Jiang, Xuntian

Cerebrospinal fluid and serum glycosphingolipid biomarkers in canine globoid cell leukodystrophy (Krabbe Disease)

犬球状细胞脑白质营养不良症(克拉伯病)脑脊液和血清糖鞘脂生物标志物

Corado, Carley R; Pinkstaff, Jason; Jiang, Xuntian; Galban, Evelyn M; Fisher, Samantha J; Scholler, Oriane; Russell, Chris; Bagel, Jessica H; ODonnell, Patricia A; Ory, Daniel S; Vite, Charles H; Bradbury, Allison M

N-acyl-O-phosphocholineserines: structures of a novel class of lipids that are biomarkers for Niemann-Pick C1 disease

N-酰基-O-磷酸胆碱丝氨酸:一类新型脂质的结构,可作为尼曼-匹克C1型疾病的生物标志物

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

Evaluation of Intrathecal Routes of Administration for Adeno-Associated Viral Vectors in Large Animals

大型动物腺相关病毒载体鞘内给药途径的评价

Hinderer, Christian; Bell, Peter; Katz, Nathan; Vite, Charles H; Louboutin, Jean-Pierre; Bote, Erin; Yu, Hongwei; Zhu, Yanqing; Casal, Margret L; Bagel, Jessica; O'Donnell, Patricia; Wang, Ping; Haskins, Mark E; Goode, Tamara; Wilson, James M

Clinical Improvement of Alpha-mannosidosis Cat Following a Single Cisterna Magna Infusion of AAV1

单次经枕大池输注AAV1后,α-甘露糖苷酶缺乏症猫的临床症状得到改善

Yoon, Sea Young; Bagel, Jessica H; O'Donnell, Patricia A; Vite, Charles H; Wolfe, John H

Temporal behavior of seizures and interictal bursts in prolonged intracranial recordings from epileptic canines

癫痫犬长期颅内记录中癫痫发作和发作间期爆发的时间行为

Ung, Hoameng; Davis, Kathryn A; Wulsin, Drausin; Wagenaar, Joost; Fox, Emily; McDonnell, John J; Patterson, Ned; Vite, Charles H; Worrell, Gregory; Litt, Brian