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

AAV-mediated gene augmentation therapy of CRB1 patient-derived retinal organoids restores the histological and transcriptional retinal phenotype

利用AAV介导的CRB1患者来源视网膜类器官基因增强疗法可恢复视网膜的组织学和转录表型

Boon, Nanda; Lu, Xuefei; Andriessen, Charlotte A; Moustakas, Ioannis; Buck, Thilo M; Freund, Christian; Arendzen, Christiaan H; Böhringer, Stefan; Boon, Camiel J F; Mei, Hailiang; Wijnholds, Jan

Defining Phenotype, Tropism, and Retinal Gene Therapy Using Adeno-Associated Viral Vectors (AAVs) in New-Born Brown Norway Rats with a Spontaneous Mutation in Crb1.

利用腺相关病毒载体(AAV)对新生褐挪威大鼠(Crb1基因自发突变)进行表型、嗜性和视网膜基因治疗的研究

Boon Nanda, Alves C Henrique, Mulder Aat A, Andriessen Charlotte A, Buck Thilo M, Quinn Peter M J, Vos Rogier M, Koster Abraham J, Jost Carolina R, Wijnholds Jan

Recombinant Adeno-Associated Viral Vectors (rAAV)-Vector Elements in Ocular Gene Therapy Clinical Trials and Transgene Expression and Bioactivity Assays

重组腺相关病毒载体(rAAV)-载体元件在眼部基因治疗临床试验及转基因表达和生物活性检测中的应用

Buck, Thilo M; Wijnholds, Jan