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

mRNA trans-splicing dual AAV vectors for (epi)genome editing and gene therapy

用于(表观)基因组编辑和基因治疗的 mRNA 反式剪接双 AAV 载体

Lisa Maria Riedmayr, Klara Sonnie Hinrichsmeyer, Stefan Bernhard Thalhammer, David Manuel Mittas, Nina Karguth, Dina Yehia Otify, Sybille Böhm, Valentin Johannes Weber, Michael David Bartoschek, Victoria Splith, Manuela Brümmer, Raphael Ferreira, Nanda Boon, Gabriele Maria Wögenstein, Christian Grim

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

AAV 介导的 CRB1 患者来源的视网膜类器官基因增强疗法可恢复组织学和转录视网膜表型

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

CRB1 is required for recycling by RAB11A+ vesicles in human retinal organoids

CRB1 是人类视网膜类器官中 RAB11A+ 囊泡回收所必需的

Thilo M Buck, Peter M J Quinn, Lucie P Pellissier, Aat A Mulder, Aldo Jongejan, Xuefei Lu, Nanda Boon, Daniëlle Koot, Hind Almushattat, Christiaan H Arendzen, Rogier M Vos, Edward J Bradley, Christian Freund, Harald M M Mikkers, Camiel J F Boon, Perry D Moerland, Frank Baas, Abraham J Koster, Jacque

Characterization and AAV-mediated CRB gene augmentation in human-derived CRB1KO and CRB1KOCRB2+/- retinal organoids

人类衍生的 CRB1KO 和 CRB1KOCRB2+/- 视网膜类器官中的表征和 AAV 介导的 CRB 基因增强

Nanda Boon, Xuefei Lu, Charlotte A Andriessen, Michaela Orlovà, Peter M J Quinn, Camiel J F Boon, Jan Wijnholds

Crumbs2 Is an Essential Slit Diaphragm Protein of the Renal Filtration Barrier

Crumbs2 是肾脏滤过屏障的必需裂孔隔膜蛋白

Annika Möller-Kerutt, Juan E Rodriguez-Gatica, Karin Wacker, Rohan Bhatia, Jan-Peter Siebrasse, Nanda Boon, Veerle Van Marck, Peter Boor, Ulrich Kubitscheck, Jan Wijnholds, Hermann Pavenstädt, Thomas Weide

CRB2 Loss in Rod Photoreceptors Is Associated with Progressive Loss of Retinal Contrast Sensitivity

杆状光感受器中的 CRB2 损失与视网膜对比敏感度的逐渐丧失有关

C Henrique Alves, Nanda Boon, Aat A Mulder, Abraham J Koster, Carolina R Jost, Jan Wijnholds

Usherin defects lead to early-onset retinal dysfunction in zebrafish

Usherin 缺陷导致斑马鱼早发性视网膜功能障碍

Margo Dona, Ralph Slijkerman, Kimberly Lerner, Sanne Broekman, Jeremy Wegner, Taylor Howat, Theo Peters, Lisette Hetterschijt, Nanda Boon, Erik de Vrieze, Nasrin Sorusch, Uwe Wolfrum, Hannie Kremer, Stephan Neuhauss, Jingjing Zang, Maarten Kamermans, Monte Westerfield, Jennifer Phillips, Erwin van W