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

Beyond Trikafta: new models to assess tissue dependent rescue of N1303K-CFTR

Trikafta 之外:评估 N1303K-CFTR 组织依赖性拯救的新模型

Pranke, Iwona; Capurro, Valeria; Chevalier, Benoit; Pesce, Emanuela; Tomati, Valeria; Pastorino, Cristina; Kelly-Aubert, Mairead; Hatton, Aurelie; Dreano, Elise; Lena, Mariateresa; Bocciardi, Renata; Zara, Federico; Pantano, Stefano; Terlizzi, Vito; Lucanto, Cristina; Costa, Stefano; Claut, Laura; Daccò, Valeria; Poli, Piercarlo; Maschio, Massimo; Fabrizzi, Benedetta; Caporelli, Nicole; Cipolli, Marco; Volpi, Sonia; Chedevergne, Frederique; Cosson, Laure; Macey, Julie; Ramel, Sophie; Weiss, Laurence; Grenet, Dominique; Le Clainche-Viala, Laurence; Douvry, Benoit; Ravoninjatovo, Bruno; Audousset, Camille; Tatopoulos, Aurélie; Richaud-Thiriez, Bénédicte; Baravalle, Melissa; Thouvenin, Guillaume; Labbé, Guillaume; Mittaine, Marie; Reix, Philippe; Durieu, Isabelle; Mankikian, Julie; Bui, Stéphanie; Nguyen-Khoa, Thao; Khoukh, Karim; Martin, Clémence; Da Silva, Jennifer; De Carli, Paola; Castellani, Carlo; Cresta, Federico; Galietta, Luis; Guillemaut, Anne; Girodon, Emmanuelle; Remus, Natacha; Bulcaen, Mathis; Ensinck, Marjolein; Zajac, Miroslaw; Carlon, Marianne; LeBihan, Jean; Burgel, Pierre-Régis; Sermet-Gaudelus, Isabelle; Hinzpeter, Alexandre; Pedemonte, Nicoletta

Protocol for functional screening of CFTR-targeted genetic therapies in patient-derived organoids using DETECTOR deep-learning-based analysis.

使用 DETECTOR 基于深度学习的分析方法对患者来源的类器官中 CFTR 靶向基因疗法进行功能筛选的方案

Bulcaen Mattijs, Liu Ronald B, Gryspeert Kasper, Thierie Sam, Ramalho Anabela S, Vermeulen François, Casadevall I Solvas Xavier, Carlon Marianne S

Cis-Regulation of the CFTR Gene in Pancreatic Cells

胰腺细胞中CFTR基因的顺式调控

Clara Blotas,Anaïs Le Nabec,Mégane Collobert,Mattijs Bulcaen,Marianne S Carlon,Claude Férec,Stéphanie Moisan

Prime editing functionally corrects cystic fibrosis-causing CFTR mutations in human organoids and airway epithelial cells

基因编辑技术能够功能性地纠正人类类器官和气道上皮细胞中导致囊性纤维化的CFTR基因突变。

Mattijs Bulcaen ,Phéline Kortleven ,Ronald B Liu ,Giulia Maule ,Elise Dreano ,Mairead Kelly ,Marjolein M Ensinck ,Sam Thierie ,Maxime Smits ,Matteo Ciciani ,Aurelie Hatton ,Benoit Chevalier ,Anabela S Ramalho ,Xavier Casadevall I Solvas ,Zeger Debyser ,François Vermeulen ,Rik Gijsbers ,Isabelle Sermet-Gaudelus ,Anna Cereseto ,Marianne S Carlon

Genetic surgery for a cystic fibrosis-causing splicing mutation

针对囊性纤维化致病剪接突变的基因手术

Bulcaen, Mattijs; Carlon, Marianne S

Nanoblades allow high-level genome editing in murine and human organoids

纳米刀可对小鼠和人类类器官进行高水平基因组编辑

Victor Tiroille, Adrien Krug, Emma Bokobza, Michel Kahi, Mattijs Bulcaen, Marjolein M Ensinck, Maarten H Geurts, Delilah Hendriks, François Vermeulen, Frédéric Larbret, Alejandra Gutierrez-Guerrero, Yu Chen, Indra Van Zundert, Susana Rocha, Anne C Rios, Louise Medaer, Rik Gijsbers, Philippe E Mangeo