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

Kelly M Podetz-Pedersen, Kanut Laoharawee, Sajya Singh, Tam T Nguyen, Miles C Smith, Alexa Temme, Karen Kozarsky, R Scott McIvor, Lalitha R Belur

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

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

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

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

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

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

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

Role of transgene regulation in ex vivo lentiviral correction of artemis deficiency

转基因调控在体外慢病毒治疗阿尔特弥斯缺陷中的作用

Megan M Multhaup, Kelly M Podetz-Pedersen, Andrea D Karlen, Erik R Olson, Roland Gunther, Nikunj V Somia, Bruce R Blazar, Morton J Cowan, R Scott McIvor

Identification of rtl1, a retrotransposon-derived imprinted gene, as a novel driver of hepatocarcinogenesis

鉴定出逆转座子来源的印迹基因 rtl1 是肝癌发生的新驱动因素

Jesse D Riordan, Vincent W Keng, Barbara R Tschida, Todd E Scheetz, Jason B Bell, Kelly M Podetz-Pedersen, Catherine D Moser, Neal G Copeland, Nancy A Jenkins, Lewis R Roberts, David A Largaespada, Adam J Dupuy

Inhibition of angiogenesis and suppression of colorectal cancer metastatic to the liver using the Sleeping Beauty Transposon System

利用睡美人转座子系统抑制血管生成并抑制结肠直肠癌转移到肝脏

Lalitha R Belur, Kelly M Podetz-Pedersen, Brent S Sorenson, Alice H Hsu, Josh B Parker, Cathy S Carlson, Daniel A Saltzman, S Ramakrishnan, R Scott McIvor

Characterization of the human artemis promoter by heterologous gene expression in vitro and in vivo

通过体外和体内异源基因表达对人类 artemis 启动子进行表征

Megan M Multhaup, Sweta Gurram, Kelly M Podetz-Pedersen, Andrea D Karlen, Debra L Swanson, Nikunj V Somia, Perry B Hackett, Morton J Cowan, R Scott McIvor

Preferential delivery of the Sleeping Beauty transposon system to livers of mice by hydrodynamic injection

通过流体动力注射将睡美人转座子系统优先输送到小鼠肝脏

Jason B Bell, Kelly M Podetz-Pedersen, Elena L Aronovich, Lalitha R Belur, R Scott McIvor, Perry B Hackett