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

Intracerebroventricular administration of a modified hexosaminidase ameliorates late-stage neurodegeneration in a GM2 mouse model

脑室内注射改良的己糖胺酶可改善 GM2 小鼠模型中的晚期神经退行性病变

Manuel E Lopez, Daniel Wendt, Roger Lawrence, Kerui Gong, Hoonsan Ong, Bryan Yip, Joseph Chen, Linley Mangini, Britta Handyside, Alexander Giaramita, Aashish Lamichhane, Melanie Lo, Vishal Agrawal, Jeremy Van Vleet, Amanda Abolhesn, Jessica B Felix, Isaac Villalpando, Vikas Bhat, Rolando De Angelis,

Prophylactic Prednisolone Promotes AAV5 Hepatocyte Transduction Through the Novel Mechanism of AAV5 Coreceptor Platelet-Derived Growth Factor Receptor Alpha Upregulation and Innate Immune Suppression

预防性泼尼松龙通过 AAV5 辅助受体血小板衍生生长因子受体 α 上调和先天免疫抑制的新机制促进 AAV5 肝细胞转导

Britta Handyside, Lening Zhang, Bridget Yates, Lin Xie, Ashrafali Mohamed Ismail, Ryan Murphy, Brian Baridon, Cheng Su, Taren Bouwman, Linley Mangini, Jorden Tahquechi, Sandra Salcido, Wesley C Minto, William T Keenan, Ioanna Ntai, Choong-Ryoul Sihn, Sherry Bullens, Stuart Bunting, Sylvia Fong

Galactokinase 1 is the source of elevated galactose-1-phosphate and cerebrosides are modestly reduced in a mouse model of classic galactosemia

半乳糖激酶 1 是半乳糖-1-磷酸升高的来源,在经典半乳糖血症小鼠模型中,脑苷脂略有减少

Linley Mangini, Roger Lawrence, Manuel E Lopez, Timothy C Graham, Christopher R Bauer, Hang Nguyen, Cheng Su, John Ramphal, Brett E Crawford, Tom A Hartl

Intracerebroventricular dosing of N-sulfoglucosamine sulfohydrolase in mucopolysaccharidosis IIIA mice reduces markers of brain lysosomal dysfunction

在粘多糖贮积症 IIIA 小鼠脑室内注射 N-磺基葡萄糖胺磺基水解酶可降低脑溶酶体功能障碍标志物

Jenna Magat, Samantha Jones, Brian Baridon, Vishal Agrawal, Hio Wong, Alexander Giaramita, Linley Mangini, Britta Handyside, Catherine Vitelli, Monica Parker, Natasha Yeung, Yu Zhou, Erno Pungor, Ilya Slabodkin, Olivia Gorostiza, Allora Aguilera, Melanie J Lo, Saida Alcozie, Terri M Christianson, Pa

Biochemical evaluation of intracerebroventricular rhNAGLU-IGF2 enzyme replacement therapy in neonatal mice with Sanfilippo B syndrome

脑室内rhNAGLU-IGF2酶替代疗法对Sanfilippo B综合征新生小鼠的生化评价

Shih-Hsin Kan, Ibrahim Elsharkawi, Steven Q Le, Heather Prill, Linley Mangini, Jonathan D Cooper, Roger Lawrence, Mark S Sands, Brett E Crawford, Patricia I Dickson

Intracerebroventricular enzyme replacement therapy with β-galactosidase reverses brain pathologies due to GM1 gangliosidosis in mice

脑室内用 β-半乳糖苷酶进行酶替代疗法可逆转小鼠因 GM1 神经节苷脂沉积症引起的脑部病变

Joseph C Chen, Amanda R Luu, Nathan Wise, Rolando De Angelis, Vishal Agrawal, Linley Mangini, Jon Vincelette, Britta Handyside, Harry Sterling, Melanie J Lo, Hio Wong, Nicole Galicia, Glenn Pacheco, Jeremy Van Vleet, Alexander Giaramita, Sylvia Fong, Sushmita M Roy, Chuck Hague, Roger Lawrence, Sher

Characterization of glycan substrates accumulating in GM1 Gangliosidosis

GM1 神经节苷脂沉积症中积累的聚糖底物的表征

Roger Lawrence, Jeremy L Van Vleet, Linley Mangini, Adam Harris, Nathan Martin, Wyatt Clark, Sanjay Chandriani, Jonathan H LeBowitz, Roberto Giugliani, Alessandra d'Azzo, Gouri Yogalingam, Brett E Crawford