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

Assessment of systemic AAV-microdystrophin gene therapy in the GRMD model of Duchenne muscular dystrophy

在杜氏肌营养不良症GRMD模型中评估全身性AAV-微型肌营养不良蛋白基因治疗

Birch, Sharla M; Lawlor, Michael W; Conlon, Thomas J; Guo, Lee-Jae; Crudele, Julie M; Hawkins, Eleanor C; Nghiem, Peter P; Ahn, Mihye; Meng, Hui; Beatka, Margaret J; Fickau, Brittany A; Prieto, Juan C; Styner, Martin A; Struharik, Michael J; Shanks, Courtney; Brown, Kristy J; Golebiowski, Diane; Bettis, Amanda K; Balog-Alvarez, Cynthia J; Clement, Nathalie; Coleman, Kirsten E; Corti, Manuela; Pan, Xiufang; Hauschka, Stephen D; Gonzalez, J Patrick; Morris, Carl A; Schneider, Joel S; Duan, Dongsheng; Chamberlain, Jeffrey S; Byrne, Barry J; Kornegay, Joe N

Toxicology and Pharmacology of an AAV Vector Expressing Codon-Optimized RPGR in RPGR-Deficient Rd9 Mice

在RPGR缺陷型Rd9小鼠中,表达密码子优化RPGR的AAV载体的毒理学和药理学研究

Song, Chunjuan; Conlon, Thomas J; Deng, Wen-Tao; Coleman, Kirsten E; Zhu, Ping; Plummer, Cayrn; Mandapati, Savitri; Van Hoosear, Mailin; Green, Kari B; Sonnentag, Peter; Sharma, Alok K; Timmers, Adrian; Robinson, Paulette M; Knop, David R; Hauswirth, William W; Chulay, Jeffrey D; Shearman, Mark S; Ye, Guo-Jie

A Preclinical Study in Rhesus Macaques for Cystic Fibrosis to Assess Gene Transfer and Transduction by AAV1 and AAV5 with a Dual-Luciferase Reporter System

一项在恒河猴中进行的囊性纤维化临床前研究,旨在利用双荧光素酶报告系统评估AAV1和AAV5的基因转移和转导效率。

Guggino, William B; Benson, Janet; Seagrave, JeanClare; Yan, Ziying; Engelhardt, John; Gao, Guangping; Conlon, Thomas J; Cebotaru, Liudmila

Safety of Intradiaphragmatic Delivery of Adeno-Associated Virus-Mediated Alpha-Glucosidase (rAAV1-CMV-hGAA) Gene Therapy in Children Affected by Pompe Disease

经膈肌递送腺相关病毒介导的α-葡萄糖苷酶(rAAV1-CMV-hGAA)基因疗法治疗庞贝病患儿的安全性

Corti, Manuela; Liberati, Cristina; Smith, Barbara K; Lawson, Lee Ann; Tuna, Ibrahim S; Conlon, Thomas J; Coleman, Kirsten E; Islam, Saleem; Herzog, Roland W; Fuller, David D; Collins, Shelley W; Byrne, Barry J

Transfer of Therapeutic Genes into Fetal Rhesus Monkeys Using Recombinant Adeno-Associated Type I Viral Vectors

利用重组腺相关I型病毒载体将治疗基因转移到胎猴体内

Conlon, Thomas J; Mah, Cathryn S; Pacak, Christina A; Rucker Henninger, Mary B; Erger, Kirsten E; Jorgensen, Marda L; Lee, C Chang I; Tarantal, Alice F; Byrne, Barry J

Evaluation of Readministration of a Recombinant Adeno-Associated Virus Vector Expressing Acid Alpha-Glucosidase in Pompe Disease: Preclinical to Clinical Planning

评估在庞贝病中再次使用表达酸性α-葡萄糖苷酶的重组腺相关病毒载体的疗效:从临床前到临床规划

Corti, Manuela; Cleaver, Brian; Clément, Nathalie; Conlon, Thomas J; Faris, Kaitlyn J; Wang, Gensheng; Benson, Janet; Tarantal, Alice F; Fuller, Davis; Herzog, Roland W; Byrne, Barry J

Regression of schwannomas induced by adeno-associated virus-mediated delivery of caspase-1

腺相关病毒介导的 caspase-1 递送诱导的神经鞘瘤消退

Prabhakar, Shilpa; Taherian, Mehran; Gianni, Davide; Conlon, Thomas J; Fulci, Giulia; Brockmann, Jillian; Stemmer-Rachamimov, Anat; Sena-Esteves, Miguel; Breakefield, Xandra O; Brenner, Gary J

Phase I/II trial of adeno-associated virus-mediated alpha-glucosidase gene therapy to the diaphragm for chronic respiratory failure in Pompe disease: initial safety and ventilatory outcomes.

腺相关病毒介导的α-葡萄糖苷酶基因疗法治疗庞贝氏症慢性呼吸衰竭的I/II期试验:初步安全性和通气结果

Smith Barbara K, Collins Shelley W, Conlon Thomas J, Mah Cathryn S, Lawson Lee Ann, Martin Anatole D, Fuller David D, Cleaver Brian D, Clément Nathalie, Phillips Dawn, Islam Saleem, Dobjia Nicole, Byrne Barry J

Preclinical toxicology and biodistribution studies of recombinant adeno-associated virus 1 human acid α-glucosidase

重组腺相关病毒1型人酸性α-葡萄糖苷酶的临床前毒理学和生物分布研究

Conlon, Thomas J; Erger, Kirsten; Porvasnik, Stacy; Cossette, Travis; Roberts, Cheryl; Combee, Lynn; Islam, Saleem; Kelley, Jeffry; Cloutier, Denise; Clément, Nathalie; Abernathy, Corinne R; Byrne, Barry J

An acidic oligopeptide displayed on AAV2 improves axial muscle tropism after systemic delivery

一种展示在AAV2上的酸性寡肽在全身给药后可改善轴向肌肉趋向性

Lee, Ni-Chung; Falk, Darin J; Byrne, Barry J; Conlon, Thomas J; Clement, Nathalie; Porvasnik, Stacy; Jorgensen, Marda L; Potter, Mark; Erger, Kirsten E; Watson, Rachael; Ghivizzani, Steven C; Chiu, Hung-Chuan; Chien, Yin-Hsiu; Hwu, Wuh-Liang