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

Intrathecal or intravenous AAV9-IDUA/RGX-111 at minimal effective dose prevents cardiac, skeletal and neurologic manifestations of murine MPS I.

鞘内或静脉注射最小有效剂量的 AAV9-IDUA/RGX-111 可预防小鼠 MPS I 的心脏、骨骼和神经系统症状

Belur Lalitha R, Huber Avery K, Mantone Hillary, Robertson Mason, Smith Miles C, Karlen Andrea D, Kitto Kelley F, Ou Li, Whitley Chester B, Braunlin Elizabeth, Furcich Justin, Lund Troy C, Seelig Davis, Fairbanks Carolyn A, Buss Nicholas, Kim Kwi Hye, McIvor R Scott

Comparative dose effectiveness of intravenous and intrathecal AAV9.CB7.hIDS, RGX-121, in mucopolysaccharidosis type II mice

静脉注射和鞘内注射 AAV9.CB7.hIDS、RGX-121 对 II 型粘多糖贮积症小鼠的剂量效果比较

Miles C Smith, Lalitha R Belur, Andrea D Karlen, Olivia Erlanson, Justin Furcich, Troy C Lund, Davis Seelig, Kelley F Kitto, Carolyn A Fairbanks, Kwi Hye Kim, Nick Buss, R Scott McIvor

Loss of early myeloid donor cell engraftment into the central nervous system with nonmyeloablative conditioning

非清髓性预处理导致早期髓系供体细胞在中枢神经系统植入失败。

Nolan, Erin E; Durose, Willa; Taghizadeh, Leyla A; King, Carina J; Gupta, Ashish O; Orchard, Paul J; Lorentson, Maggie; Braaten, Kai; Furcich, Justin W; Lund, Troy C

Generation and characterization of an immunodeficient mouse model of mucopolysaccharidosis type II

粘多糖贮积症 II 型免疫缺陷小鼠模型的建立及鉴定

Miles C Smith, Lalitha R Belur, Andrea D Karlen, Kelly Podetz-Pedersen, Olivia Erlanson, Kanut Laoharawee, Justin Furcich, Troy C Lund, Yun You, Davis Seelig, Beau R Webber, R Scott McIvor

Phenotypic Correction of Murine Mucopolysaccharidosis Type II by Engraftment of Ex Vivo Lentiviral Vector-Transduced Hematopoietic Stem and Progenitor Cells

通过体外慢病毒载体转导的造血干细胞和祖细胞植入对小鼠粘多糖贮积症 II 型进行表型校正

Miles C Smith, Lalitha R Belur, Andrea D Karlen, Olivia Erlanson, Kelly M Podetz-Pedersen, Jessica McKenzie, Jenn Detellis, Khatuna Gagnidze, Geoffrey Parsons, Nicholas Robinson, Shelby Labarre, Saumil Shah, Justin Furcich, Troy C Lund, Hsing-Chen Tsai, R Scott McIvor, Melissa Bonner

Glycoprotein nonmetastatic melanoma protein B (GNMPB) as a novel biomarker for cerebral adrenoleukodystrophy

糖蛋白非转移性黑色素瘤蛋白 B (GNMPB) 作为脑肾上腺脑白质营养不良的新型生物标志物

Leyla A Taghizadeh, Carina J King, David R Nascene, Ashish O Gupta, Paul J Orchard, LeeAnn Higgins, Todd W Markowski, Erin E Nolan, Justin W Furcich, Troy C Lund

An irradiated marrow niche reveals a small noncollagenous protein mediator of homing, dermatopontin

受辐射的骨髓微环境揭示了一种小型非胶原蛋白归巢介质——皮桥蛋白

Ashley C Kramer, Yuliana Astuti, Alexis Elfstrum, Michael J Lehrke, Jakub Tolar, Bruce R Blazar, Amanda L Blake, Mandy E Taisto, Justin W Furcich, Erin E Nolan, Wilaiwan W Durose, Beau R Webber, Athena Geisness, David K Wood, Troy C Lund