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

Establishing a GMP-compliant manufacturing process and phase-appropriate analytics for early development of a FiCAR T-cell product with a novel CAR spacer.

建立符合 GMP 标准的生产工艺和阶段性分析方法,用于早期开发具有新型 CAR 间隔物的 FiCAR T 细胞产品

Luostarinen Annu, Vuorela Arja, Kerkelä Erja, Patrikoski Mimmi, Kotovuori Annika, Koski Jan, Ahoniemi Jonna, Lähteenmäki Kaarina, Lehtisalo Jenni, Oja Terhi, Paavilainen Henrik, Autio Anu, Nyman Marie, Nikoskelainen Veera, Kergourlay Virginie, Elbasani Endrit, van Veen Bert, Thotakura Anil, Monzo Hector, Ojala Päivi M, Korhonen Matti, Hongisto Heidi, Laitinen Anita

Correction of pseudoexon splicing caused by a novel intronic dysferlin mutation.

纠正由新型内含子 dysferlin 突变引起的假外显子剪接

Dominov Janice A, Uyan Özgün, McKenna-Yasek Diane, Nallamilli Babi Ramesh Reddy, Kergourlay Virginie, Bartoli Marc, Levy Nicolas, Hudson Judith, Evangelista Teresinha, Lochmuller Hanns, Krahn Martin, Rufibach Laura, Hegde Madhuri, Brown Robert H Jr

Comment on: A novel dysferlin-mutant pseudoexon bypassed with antisense oligonucleotides

评论:利用反义寡核苷酸绕过一种新型的dysferlin突变假外显子

Kergourlay, Virginie; Blandin, Gaëlle; Blanck, Véronique; Lévy, Nicolas; Bartoli, Marc; Krahn, Martin

Exon 32 Skipping of Dysferlin Rescues Membrane Repair in Patients' Cells

外显子32跳跃导致Dysferlin基因表达异常,从而挽救患者细胞的膜修复功能。

Barthélémy, Florian; Blouin, Cédric; Wein, Nicolas; Mouly, Vincent; Courrier, Sébastien; Dionnet, Eugénie; Kergourlay, Virginie; Mathieu, Yves; Garcia, Luis; Butler-Browne, Gillian; Lamaze, Christophe; Lévy, Nicolas; Krahn, Martin; Bartoli, Marc