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

CRISPR and gene editing technologies for bleeding disorders

CRISPR和基因编辑技术在出血性疾病治疗中的应用

VandenDriessche, Thierry; Janssens, Mathias; Chuah, Marinee K

Treatment of infantile-onset Pompe disease in a rat model with muscle-directed AAV gene therapy

肌肉定向 AAV 基因治疗治疗大鼠模型中的婴儿型庞贝氏症

Sergio Muñoz, Joan Bertolin, Veronica Jimenez, Maria Luisa Jaén, Miquel Garcia, Anna Pujol, Laia Vilà, Victor Sacristan, Elena Barbon, Giuseppe Ronzitti, Jihad El Andari, Warut Tulalamba, Quang Hong Pham, Jesus Ruberte, Thierry VandenDriessche, Marinee K Chuah, Dirk Grimm, Federico Mingozzi, Fatima

Comprehensive analysis of off-target and on-target effects resulting from liver-directed CRISPR-Cas9-mediated gene targeting with AAV vectors

利用AAV载体进行肝脏靶向CRISPR-Cas9介导的基因靶向治疗所产生的脱靶效应和靶向效应的综合分析

Singh, Kshitiz; Fronza, Raffaele; Evens, Hanneke; Chuah, Marinee K; VandenDriessche, Thierry

Comprehensive transcriptome-wide analysis of spliceopathy correction of myotonic dystrophy using CRISPR-Cas9 in iPSCs-derived cardiomyocytes

使用 CRISPR-Cas9 对 iPSC 衍生的心肌细胞中的肌强直性营养不良症进行全面的转录组范围分析

Sumitava Dastidar, Debanjana Majumdar, Jaitip Tipanee, Kshitiz Singh, Arnaud F Klein, Denis Furling, Marinee K Chuah, Thierry VandenDriessche

Universal allogeneic CAR T cells engineered with Sleeping Beauty transposons and CRISPR-CAS9 for cancer immunotherapy

利用 Sleeping Beauty 转座子和 CRISPR-Cas9 技术改造的通用同种异体 CAR-T 细胞用于癌症免疫治疗

Tipanee, Jaitip; Samara-Kuko, Ermira; Gevaert, Thierry; Chuah, Marinee K; VandenDriessche, Thierry

Hemophilia "A" gene therapy: Lost in translation

血友病A基因疗法:翻译中的误区

VandenDriessche, Thierry; Chuah, Marinee K

Identification of a myotropic AAV by massively parallel in vivo evaluation of barcoded capsid variants

通过大规模并行体内评估条形码衣壳变体来鉴定肌向性 AAV

Jonas Weinmann, Sabrina Weis, Josefine Sippel, Warut Tulalamba, Anca Remes, Jihad El Andari, Anne-Kathrin Herrmann, Quang H Pham, Christopher Borowski, Susanne Hille, Tanja Schönberger, Norbert Frey, Martin Lenter, Thierry VandenDriessche, Oliver J Müller, Marinee K Chuah, Thorsten Lamla, Dirk Grimm

Validation of miR-20a as a Tumor Suppressor Gene in Liver Carcinoma Using Hepatocyte-Specific Hyperactive piggyBac Transposons

利用肝细胞特异性高活性 piggyBac 转座子验证 miR-20a 作为肝癌抑癌基因的功能

Tipanee, Jaitip; Di Matteo, Mario; Tulalamba, Warut; Samara-Kuko, Ermira; Keirsse, Jiri; Van Ginderachter, Jo A; Chuah, Marinee Khim; VandenDriessche, Thierry

Genetic and Epigenetic Modification of Rat Liver Progenitor Cells via HNF4α Transduction and 5' Azacytidine Treatment: An Integrated miRNA and mRNA Expression Profile Analysis.

通过 HNF4α 转导和 5' 氮杂胞苷处理对大鼠肝脏祖细胞进行遗传和表观遗传修饰:miRNA 和 mRNA 表达谱的综合分析

Bolleyn Jennifer, Rombaut Matthias, Nair Nisha, Branson Steven, Heymans Anja, Chuah Marinee, VandenDriessche Thierry, Rogiers Vera, De Kock Joery, Vanhaecke Tamara

Efficient In Vivo Liver-Directed Gene Editing Using CRISPR/Cas9

使用 CRISPR/Cas9 进行有效的体内肝脏定向基因编辑

Kshitiz Singh, Hanneke Evens, Nisha Nair, Melvin Y Rincón, Shilpita Sarcar, Ermira Samara-Kuko, Marinee K Chuah, Thierry VandenDriessche