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

Neurologic Recovery in MPS I and MPS II Mice by AAV9-Mediated Gene Transfer to the CNS After the Development of Cognitive Dysfunction

认知功能障碍发生后，通过 AAV9 介导的中枢神经系统基因转移，MPS I 和 MPS II 小鼠的神经功能得以恢复

期刊:Human Gene Therapy
影响因子:3.9
doi:10.1089/hum.2022.162
Kelly M Podetz-Pedersen, Kanut Laoharawee, Sajya Singh, Tam T Nguyen, Miles C Smith, Alexa Temme, Karen Kozarsky, R Scott McIvor, Lalitha R Belur
信号转导
神经
发表日期：2023
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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

期刊:Molecular Genetics and Metabolism
影响因子:3.7
doi:10.1016/j.ymgme.2023.107539
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
免疫
Mouse
发表日期：2023
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Non-invasive intravenous administration of AAV9 transducing iduronate sulfatase leads to global metabolic correction and prevention of neurologic deficits in a mouse model of Hunter syndrome

通过非侵入性静脉注射 AAV9 转导艾杜糖醛酸硫酸酯酶，可纠正亨特综合征小鼠的整体代谢并预防神经系统缺陷

期刊:Molecular Genetics and Metabolism Reports
影响因子:1.8
doi:10.1016/j.ymgmr.2023.100956
Kanut Laoharawee, Kelly M Podetz-Pedersen, Tam T Nguyen, Sajya M Singh, Miles C Smith, Lalitha R Belur, Walter C Low, Karen F Kozarsky, R Scott McIvor
代谢
信号转导
神经
Neuroscience
Mouse
发表日期：2023
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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

期刊:Human Gene Therapy
影响因子:3.9
doi:10.1089/hum.2022.141
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
信号转导
发育与干细胞
微生物学
干细胞
发表日期：2022
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Comparative Effectiveness of Intracerebroventricular, Intrathecal, and Intranasal Routes of AAV9 Vector Administration for Genetic Therapy of Neurologic Disease in Murine Mucopolysaccharidosis Type I

比较脑室内、鞘内和鼻内三种途径给予AAV9载体治疗小鼠I型粘多糖贮积症神经系统疾病的基因治疗效果

期刊:Frontiers in Molecular Neuroscience
影响因子:3.8
doi:10.3389/fnmol.2021.618360
Belur, Lalitha R; Romero, Megan; Lee, Junggu; Podetz-Pedersen, Kelly M; Nan, Zhenhong; Riedl, Maureen S; Vulchanova, Lucy; Kitto, Kelley F; Fairbanks, Carolyn A; Kozarsky, Karen F; Orchard, Paul J; Frey, William H 2nd; Low, Walter C; McIvor, R Scott
基因治疗
神经科学
发表日期：2021
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Intravenous delivery for treatment of mucopolysaccharidosis type I: A comparison of AAV serotypes 9 and rh10

静脉注射治疗I型粘多糖贮积症：AAV血清型9和rh10的比较

期刊:Molecular Genetics and Metabolism Reports
影响因子:1.9
doi:10.1016/j.ymgmr.2020.100604
Belur, Lalitha R; Podetz-Pedersen, Kelly M; Tran, Thuy An; Mesick, Joshua A; Singh, Nathaniel M; Riedl, Maureen; Vulchanova, Lucy; Kozarsky, Karen F; McIvor, R Scott
发表日期：2020
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ZFN-Mediated In Vivo Genome Editing Corrects Murine Hurler Syndrome

ZFN介导的体内基因组编辑可纠正小鼠赫勒氏综合征

期刊:Molecular Therapy
影响因子:12.1
doi:10.1016/j.ymthe.2018.10.018
Li Ou，Russell C DeKelver，Michelle Rohde，Susan Tom，Robert Radeke，Susan J St Martin，Yolanda Santiago，Scott Sproul，Michael J Przybilla，Brenda L Koniar，Kelly M Podetz-Pedersen，Kanut Laoharawee，Renee D Cooksley，Kathleen E Meyer，Michael C Holmes，R Scott McIvor，Thomas Wechsler，Chester B Whitley
发表日期：2019
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Dose-Dependent Prevention of Metabolic and Neurologic Disease in Murine MPS II by ZFN-Mediated In Vivo Genome Editing

ZFN 介导的体内基因组编辑对小鼠 MPS II 中的代谢和神经系统疾病进行剂量依赖性预防

期刊:Molecular Therapy
影响因子:12.1
doi:10.1016/j.ymthe.2018.03.002
Kanut Laoharawee, Russell C DeKelver, Kelly M Podetz-Pedersen, Michelle Rohde, Scott Sproul, Hoang-Oanh Nguyen, Tam Nguyen, Susan J St Martin, Li Ou, Susan Tom, Robert Radeke, Kathleen E Meyer, Michael C Holmes, Chester B Whitley, Thomas Wechsler, R Scott McIvor
代谢
信号转导
神经
发表日期：2018
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Intranasal Adeno-Associated Virus Mediated Gene Delivery and Expression of Human Iduronidase in the Central Nervous System: A Noninvasive and Effective Approach for Prevention of Neurologic Disease in Mucopolysaccharidosis Type I

鼻内腺相关病毒介导的人艾杜糖醛酸酶基因递送及在中枢神经系统中的表达：一种预防I型粘多糖贮积症神经系统疾病的非侵入性有效方法

期刊:Human Gene Therapy
影响因子:4
doi:10.1089/hum.2017.187
Belur, Lalitha R; Temme, Alexa; Podetz-Pedersen, Kelly M; Riedl, Maureen; Vulchanova, Lucy; Robinson, Nicholas; Hanson, Leah R; Kozarsky, Karen F; Orchard, Paul J; Frey, William H 2nd; Low, Walter C; McIvor, R Scott
Human
发表日期：2017
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A Broad Range of Dose Optima Achieve High-level, Long-term Gene Expression After Hydrodynamic Delivery of Sleeping Beauty Transposons Using Hyperactive SB100x Transposase

利用高活性SB100x转座酶进行水动力递送睡美人转座子后，多种剂量组合均可实现高水平、长期的基因表达。

期刊:Molecular Therapy-Nucleic Acids
影响因子:6.1
doi:10.1038/mtna.2015.54
Podetz-Pedersen, Kelly M; Olson, Erik R; Somia, Nikunj V; Russell, Stephen J; McIvor, R Scott
发表日期：2016
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