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

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

Comparative impact of AAV and enzyme replacement therapy on respiratory and cardiac function in adult Pompe mice

AAV 和酶替代疗法对成年庞贝氏症小鼠呼吸和心脏功能的比较影响

Falk, Darin J; Soustek, Meghan S; Todd, Adrian Gary; Mah, Cathryn S; Cloutier, Denise A; Kelley, Jeffry S; Clement, Nathalie; Fuller, David D; Byrne, Barry J

Adeno-associated virus-mediated gene therapy for metabolic myopathy

腺相关病毒介导的基因疗法治疗代谢性肌病

Mah, Cathryn S; Soustek, Meghan S; Todd, A Gary; McCall, Angela; Smith, Barbara K; Corti, Manuela; Falk, Darin J; Byrne, Barry 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

Intrapleural administration of AAV9 improves neural and cardiorespiratory function in Pompe disease

胸膜内注射AAV9可改善庞贝病患者的神经和心肺功能

Falk, Darin J; Mah, Cathryn S; Soustek, Meghan S; Lee, Kun-Ze; Elmallah, Mai K; Cloutier, Denise A; Fuller, David D; Byrne, Barry J

Gene therapy approaches for lysosomal storage disease: next-generation treatment

溶酶体贮积症的基因治疗方法:下一代疗法

Byrne, Barry J; Falk, Darin J; Clément, Nathalie; Mah, Cathryn S

Characterization of a transgenic short hairpin RNA-induced murine model of Tafazzin deficiency

对转基因短发夹RNA诱导的Tafazzin缺陷小鼠模型进行表征

Soustek, Meghan S; Falk, Darin J; Mah, Cathryn S; Toth, Matthew J; Schlame, Michael; Lewin, Alfred S; Byrne, Barry J

Pompe disease gene therapy

庞贝病基因治疗

Byrne, Barry J; Falk, Darin J; Pacak, Christina A; Nayak, Sushrusha; Herzog, Roland W; Elder, Melissa E; Collins, Shelley W; Conlon, Thomas J; Clement, Nathalie; Cleaver, Brian D; Cloutier, Denise A; Porvasnik, Stacy L; Islam, Saleem; Elmallah, Mai K; Martin, Anatole; Smith, Barbara K; Fuller, David D; Lawson, Lee Ann; Mah, Cathryn S

Glycogen storage disease type Ia in canines: a model for human metabolic and genetic liver disease

犬类糖原贮积症 Ia 型:人类代谢性和遗传性肝病的模型

Specht, Andrew; Fiske, Laurie; Erger, Kirsten; Cossette, Travis; Verstegen, John; Campbell-Thompson, Martha; Struck, Maggie B; Lee, Young Mok; Chou, Janice Y; Byrne, Barry J; Correia, Catherine E; Mah, Cathryn S; Weinstein, David A; Conlon, Thomas J

Gel-mediated delivery of AAV1 vectors corrects ventilatory function in Pompe mice with established disease

通过凝胶介导递送AAV1载体可纠正已确诊庞贝氏症小鼠的呼吸功能。

Mah, Cathryn S; Falk, Darin J; Germain, Sean A; Kelley, Jeffry S; Lewis, Melissa A; Cloutier, Denise A; DeRuisseau, Lara R; Conlon, Thomas J; Cresawn, Kerry O; Fraites, Thomas J Jr; Campbell-Thompson, Martha; Fuller, David D; Byrne, Barry J