Chandler Randy J相关文献与解析，生知库 | 链接生命科学的每一步

Hemmingsen Madeline G, Zhang Guo-Fang, Ma Yunhan, Marchuk Hannah, Patel Kalyani R, Chen Tong, Li Xinning, Chapman Mark, Collias Sabrina, Lopez-Terrada Dolores H, Beasley James, Stiles Ashlee R, Chandler Randy J, Venditti Charles P, Young Sarah P, Barzi Mercedes, Bissig-Choisat Beatrice, Krafte Doug, Newgard Christopher B, Bissig Karl-Dimiter

线粒体

发表日期：2025

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Adeno-Associated Virus Gene Therapy Development: Early Planning and Regulatory Considerations to Advance the Platform Vector Gene Therapy Program

腺相关病毒基因治疗开发：推进平台载体基因治疗项目的早期规划和监管考量

期刊:Human Gene Therapy

影响因子:4

doi:10.1089/hum.2024.230

Lomash, Richa Madan; Dehdashti, Jean; Shchelochkov, Oleg A; Chandler, Randy J; Li, Lina; Manoli, Irini; Sloan, Jennifer L; Terse, Pramod; Xu, Xin; Saade, Dimah; Stan, Rodica; Brooks, Philip J; Lo, Donald C; Bönnemann, Carsten G; Venditti, Charles P; Pariser, Anne R; Ottinger, Elizabeth A

Determining recombinant AAV capsid extracellular and intracellular biodistribution by dual radioisotope labeling

利用双放射性同位素标记法测定重组AAV衣壳的细胞外和细胞内生物分布

期刊:Molecular Therapy-Methods & Clinical Development

影响因子:4.7

doi:10.1016/j.omtm.2024.101373

From Puppies to adults: In vivo editing of hepatocytes in a canine model of glycogen storage disease type Ia

从幼犬到成犬：在犬类Ia型糖原贮积症模型中对肝细胞进行体内编辑

期刊:Molecular Therapy-Methods & Clinical Development

影响因子:4.7

doi:10.1016/j.omtm.2023.04.006

Successfully Navigating Food and Drug Administration Orphan Drug and Rare Pediatric Disease Designations for AAV9-hPCCA Gene Therapy: The National Institutes of Health Platform Vector Gene Therapy Experience

成功获得美国食品药品监督管理局 (FDA) 孤儿药和罕见儿科疾病认定：AAV9-hPCCA 基因疗法的美国国立卫生研究院平台载体基因疗法经验

期刊:Human Gene Therapy

影响因子:4

doi:10.1089/hum.2022.232

Lomash, Richa Madan; Shchelochkov, Oleg; Chandler, Randy J; Venditti, Charles P; Pariser, Anne R; Ottinger, Elizabeth A

PCCA

发表日期：2023

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Evaluating the state of the science for adeno-associated virus integration: An integrated perspective

评估腺相关病毒整合的科学现状：一个综合视角

期刊:Molecular Therapy

影响因子:12

doi:10.1016/j.ymthe.2022.06.004

Sabatino, Denise E; Bushman, Frederic D; Chandler, Randy J; Crystal, Ronald G; Davidson, Beverly L; Dolmetsch, Ricardo; Eggan, Kevin C; Gao, Guangping; Gil-Farina, Irene; Kay, Mark A; McCarty, Douglas M; Montini, Eugenio; Ndu, Adora; Yuan, Jing

微生物学

发表日期：2022

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Enhanced Efficacy and Increased Long-Term Toxicity of CNS-Directed, AAV-Based Combination Therapy for Krabbe Disease

针对克拉伯病的中枢神经系统靶向AAV联合疗法疗效增强，但长期毒性增加

期刊:Molecular Therapy

影响因子:12

doi:10.1016/j.ymthe.2020.12.031

Li, Yedda; Miller, Christopher A; Shea, Lauren K; Jiang, Xuntian; Guzman, Miguel A; Chandler, Randy J; Ramakrishnan, Sai M; Smith, Stephanie N; Venditti, Charles P; Vogler, Carole A; Ory, Daniel S; Ley, Timothy J; Sands, Mark S

ImmTOR nanoparticles enhance AAV transgene expression after initial and repeat dosing in a mouse model of methylmalonic acidemia

在甲基丙二酸血症小鼠模型中，ImmTOR纳米颗粒在首次和重复给药后可增强AAV转基因的表达。

期刊:Molecular Therapy-Methods & Clinical Development

影响因子:4.7

doi:10.1016/j.omtm.2021.06.015

Ilyinskii, Petr O; Michaud, Alicia M; Rizzo, Gina L; Roy, Christopher J; Leung, Sheldon S; Elkins, Stephanie L; Capela, Teresa; Chowdhury, Aparajita; Li, Lina; Chandler, Randy J; Manoli, Irini; Andres-Mateos, Eva; Johnston, Lloyd P M; Vandenberghe, Luk H; Venditti, Charles P; Kishimoto, Takashi Kei

mTOR

发表日期：2021

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Low incidence of hepatocellular carcinoma in mice and cats treated with systemic adeno-associated viral vectors.

用全身性腺相关病毒载体治疗的小鼠和猫中肝细胞癌的发生率较低

期刊:Molecular Therapy-Methods & Clinical Development

影响因子:4.7

doi:10.1016/j.omtm.2020.11.015

Ferla Rita, Alliegro Marialuisa, Dell'Anno Margherita, Nusco Edoardo, Cullen John M, Smith Stephanie N, Wolfsberg Tyra G, O'Donnell Patricia, Wang Ping, Nguyen Anh-Dao, Chandler Randy J, Chen Zelin, Burgess Shawn M, Vite Charles H, Haskins Mark E, Venditti Charles P, Auricchio Alberto

The Platform Vector Gene Therapies Project: Increasing the Efficiency of Adeno-Associated Virus Gene Therapy Clinical Trial Startup

平台载体基因疗法项目：提高腺相关病毒基因疗法临床试验启动效率

期刊:Human Gene Therapy

影响因子:4

doi:10.1089/hum.2020.259

Brooks, Philip J; Ottinger, Elizabeth A; Portero, Deanna; Lomash, Richa Madan; Alimardanov, Asaf; Terse, Pramod; Xu, Xin; Chandler, Randy J; Geist Hauserman, Janelle; Esposito, Eric; Bönnemann, Carsten G; Venditti, Charles P; Austin, Christopher P; Pariser, Anne; Lo, Donald C

微生物学

发表日期：2020

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Dissecting the effect of mitochondrial BCAT inhibition in methylmalonic acidemia.

剖析线粒体 BCAT 抑制对甲基丙二酸血症的影响。

Adeno-Associated Virus Gene Therapy Development: Early Planning and Regulatory Considerations to Advance the Platform Vector Gene Therapy Program

腺相关病毒基因治疗开发：推进平台载体基因治疗项目的早期规划和监管考量

Determining recombinant AAV capsid extracellular and intracellular biodistribution by dual radioisotope labeling

利用双放射性同位素标记法测定重组AAV衣壳的细胞外和细胞内生物分布

From Puppies to adults: In vivo editing of hepatocytes in a canine model of glycogen storage disease type Ia

从幼犬到成犬：在犬类Ia型糖原贮积症模型中对肝细胞进行体内编辑

Successfully Navigating Food and Drug Administration Orphan Drug and Rare Pediatric Disease Designations for AAV9-hPCCA Gene Therapy: The National Institutes of Health Platform Vector Gene Therapy Experience

成功获得美国食品药品监督管理局 (FDA) 孤儿药和罕见儿科疾病认定：AAV9-hPCCA 基因疗法的美国国立卫生研究院平台载体基因疗法经验

Evaluating the state of the science for adeno-associated virus integration: An integrated perspective

评估腺相关病毒整合的科学现状：一个综合视角

Enhanced Efficacy and Increased Long-Term Toxicity of CNS-Directed, AAV-Based Combination Therapy for Krabbe Disease

针对克拉伯病的中枢神经系统靶向AAV联合疗法疗效增强，但长期毒性增加

ImmTOR nanoparticles enhance AAV transgene expression after initial and repeat dosing in a mouse model of methylmalonic acidemia

在甲基丙二酸血症小鼠模型中，ImmTOR纳米颗粒在首次和重复给药后可增强AAV转基因的表达。

Low incidence of hepatocellular carcinoma in mice and cats treated with systemic adeno-associated viral vectors.

用全身性腺相关病毒载体治疗的小鼠和猫中肝细胞癌的发生率较低

The Platform Vector Gene Therapies Project: Increasing the Efficiency of Adeno-Associated Virus Gene Therapy Clinical Trial Startup

平台载体基因疗法项目：提高腺相关病毒基因疗法临床试验启动效率