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

Impact of CFTR Modulator Concentrations on Clinical Response in Cystic Fibrosis

CFTR调节剂浓度对囊性纤维化临床反应的影响

Chalamalla, Ashritha R; Baker, Elizabeth; Ryan, Kevin J; Dowell, Alexander; Natt, Jennifer R; Zemanick, Edith T; Konstan, Michael W; Mayer-Hamblett, Nicole; Acosta, Edward P; Guimbellot, Jennifer S

The European Medicines Agency's review of elexacaftor/tezacaftor/ivacaftor: extending its use to all people with cystic fibrosis aged 2 years and older who do not have two class I CFTR variants

欧洲药品管理局对 elexacaftor/tezacaftor/ivacaftor 的审查:将其使用范围扩大到所有 2 岁及以上、不携带两个 I 类 CFTR 变异体的囊性纤维化患者。

Drai, Catherine; van der Woude, Hanneke J; Lexmond, Anne J; van den Hoorn, Tineke; Mol, Peter G M; Leacy, Finbarr P; Przybyszewska, Agnieszka; Sepodes, Bruno; Castelnovo, Thomas

Advances in cystic fibrosis: CFTR modulator triple combinations

囊性纤维化治疗进展:CFTR调节剂三联疗法

Burgel, Pierre-Régis; Mall, Marcus A

Elexacaftor/Tezacaftor/Ivacaftor for Cystic Fibrosis and Rare CFTR Variants: In Vitro Translation to a Phase 3, Double-Blind, Randomized, Placebo-controlled Trial and Real-World Study

Elexacaftor/Tezacaftor/Ivacaftor 用于囊性纤维化和罕见 CFTR 变异:体外研究向 3 期双盲、随机、安慰剂对照试验和真实世界研究的转化

Castellani, Carlo; Mondejar-Lopez, Pedro; Van Goor, Fredrick; Quon, Bradley S; Alghisi, Federico; Fabrizzi, Benedetta; Ramsey, Bonnie; Taylor-Cousar, Jennifer L; McKone, Edward F; Tullis, Elizabeth; Weinstock, Tanya G; Tan, Vincent; Ahluwalia, Neil; Mahic, Milada; Liu, Lingyun; Saayman, Sheena; Altshuler, David; Waltz, David; Mall, Marcus A; Fajac, Isabelle

LUNAR LNP delivery of CFTR mRNA restores channel function and improves mucociliary clearance in ferret cystic fibrosis airways

LUNAR LNP递送CFTR mRNA可恢复雪貂囊性纤维化气道中的通道功能并改善黏液纤毛清除功能。

Liu, Xiaoming; Campos-Gomez, Javier; Luo, Meihui; Ma, Jia; Chen, Han-Chung; Shukla, Sourabh; Wu, Shuang; Snow, Guy; Pei, Yihua; Boehme, Stefen A; Geller, David E; Rowe, Steve M; Engelhardt, John F

Who will win the race for the first CFTR gene replacement therapy?

谁将赢得首个CFTR基因替代疗法的竞赛?

Brewington, John J; Mall, Marcus A; Maeda, Yutaka

Beyond CFTR: Ivacaftor's role in restoring cellular redox balance and preventing ferroptosis.

超越 CFTR:伊伐卡托在恢复细胞氧化还原平衡和防止铁死亡中的作用。

Rubin Michela, Artusi Ilaria, Rossetto Monica, Gucciardi Antonina, Di Paolo Maria Luisa, Bosello Travain Valentina, Zeppilli Davide, Orian Laura, Miotto Giovanni, Friedmann Angeli José Pedro, Cozza Giorgio

CFTR modulators partially restore the epithelial interferome in Aspergillus infection to improve clinical outcome

CFTR调节剂可部分恢复曲霉菌感染中的上皮细胞干扰素组,从而改善临床疗效。

Laverty, Sarah L; Felton, Imogen; Casey, Michelle; Hopwood, Benjamin; Simmonds, Nicholas; Armstrong-James, Darius; Shah, Anand; Kelleher, Peter

Single-molecule dissection of CFTR folding defects and pharmacological rescue

单分子层面解析CFTR折叠缺陷及药物补救

Kim, Sang Ah; Levring, Jesper; Chen, Jue; Yoon, Tae-Young

Chronic and acute modulator treatment restore wild-type-like activity and stability to the primary cystic fibrosis-causing CFTR variant

慢性及急性调节剂治疗可使导致囊性纤维化的原发性 CFTR 变体恢复到野生型水平的活性和稳定性。

Rodrat, Mayuree; Charlick, James N; Sheppard, David N