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

A novel dual-plasmid platform provides scalable transfection yielding improved productivity and packaging across multiple AAV serotypes and genomes

一种新型双质粒平台可实现可扩展的转染,从而提高多种AAV血清型和基因组的生产力和包装效率。

van Lieshout, Laura P; Rubin, Miranda; Costa-Grant, Katrina; Ota, Stacy; Golebiowski, Diane; Panico, Troy; Wiberg, Eli; Szymczak, Klaudia; Gilmore, Richard; Stanvick, Marissa; Burnham, Brenda; Gagnon, Jeff; Iwuchukwu, Ifeyinwa; Yang, Guang; Ghazi, Iraj; Meola, Alex; Dickerson, Ryan; Thiers, Thomas; Mustich, Luke; Hayes, April; Rivas, Israel; Lotterhand, Jason; Avila, Nancy; McGivney, James; Yin, Jin; Kelly, Tim

Single Systemic Administration of a Gene Therapy Leading to Disease Treatment in Metachromatic Leukodystrophy Arsa Knock-Out Mice

单次系统性基因疗法治疗异染性脑白质营养不良症 Arsa 基因敲除小鼠的疾病

Thia St Martin, Tania A Seabrook, Katherine Gall, Jenn Newman, Nancy Avila, April Hayes, Monicah Kivaa, Jason Lotterhand, Michael Mercaldi, Kruti Patel, Israel J Rivas, Stephen Woodcock, Teresa L Wright, Albert B Seymour, Omar L Francone, Jacinthe Gingras

Natural variations in AAVHSC16 significantly reduce liver tropism and maintain broad distribution to periphery and CNS

AAVHSC16 的自然变异显著降低了肝脏趋向性,并保持了对外周和中枢神经系统的广泛分布

Laura J Smith, Lindsay A Schulman, Samantha Smith, Laura Van Lieshout, Carmen M Barnes, Liana Behmoiras, Meghan Scarpitti, Monicah Kivaa, Khanh L Duong, Ludo O Benard, Jeff L Ellsworth, Nancy Avila, Deiby Faulkner, April Hayes, Jason Lotterhand, Jose Israel Rivas, Arnold V Sengooba, Alec Tzianabos, 

Sustained Correction of a Murine Model of Phenylketonuria following a Single Intravenous Administration of AAVHSC15-PAH

单次静脉注射AAVHSC15-PAH后,苯丙酮尿症小鼠模型得到持续纠正

Ahmed, Seemin S; Rubin, Hillard; Wang, Minglun; Faulkner, Deiby; Sengooba, Arnold; Dollive, Serena N; Avila, Nancy; Ellsworth, Jeff L; Lamppu, Diana; Lobikin, Maria; Lotterhand, Jason; Adamson-Small, Laura; Wright, Teresa; Seymour, Albert; Francone, Omar L

Nonclinical comparability studies of recombinant human arylsulfatase A addressing manufacturing process changes

重组人芳基硫酸酯酶 A 的非临床可比性研究涉及制造工艺的变化

Teresa Wright, Aiqun Li, Jason Lotterhand, Anne-Renee Graham, Yan Huang, Nancy Avila, Jing Pan

CNS penetration of intrathecal-lumbar idursulfase in the monkey, dog and mouse: implications for neurological outcomes of lysosomal storage disorder

鞘内腰椎注射艾杜硫酶对猴、狗和小鼠中枢神经系统的渗透:对溶酶体贮积症神经系统预后的影响

Calias, Pericles; Papisov, Mikhail; Pan, Jing; Savioli, Nancy; Belov, Vasily; Huang, Yan; Lotterhand, Jason; Alessandrini, Mary; Liu, Nan; Fischman, Alan J; Powell, Jan L; Heartlein, Michael W