Results 141 to 150 of about 12,522 (293)

Quantitative Systems Toxicology Predicts Ivacaftor‐Induced Oxidative Stress Contributes to CFTR Modulator Hepatotoxicity

Clinical Pharmacology &Therapeutics, Volume 119, Issue 1, Page 208-218, January 2026.
Cystic fibrosis (CF) is a chronic hereditary disease that affects tens of thousands of people worldwide. The introduction of CFTR modulator therapies such as elexacaftor/tezacaftor/ivacaftor (ETI) has significantly improved the quality of life of people with CF.
Alan Shi, Cole Cornwell, Kyunghee Yang, Paul M. Beringer   +3 more
wiley   +1 more source

Single‐nucleus RNA sequencing reveals the distinct heterogeneity of primary pulmonary sarcomas (PPS) and pulmonary sarcomatoid carcinoma (PSC)

Clinical and Translational Medicine, Volume 16, Issue 1, January 2026.
Single‐nucleus RNA sequencing (snRNA‐seq) maps the cellular diversity of pulmonary sarcomatoid carcinoma (PSC) and primary pulmonary sarcomas (PPS). AT2 cells, a major source of epithelial cells in PSC, underwent malignant transformation primarily through epithelial–mesenchymal transition, suggesting AT2 cells may serve as the origin of PSC.
Jianfei Zhu, Yanlu Xiong, Shouzheng Ma, Jun Wei, Jiakuan Chen, Wenchen Wang, Qianqian Duan, Qin Zhang, Dongsheng Chen, Wanglong Deng, Tao Jiang, Jie Lei   +11 more
wiley   +1 more source

The validated French CFAbd‐Score reveals a lower burden of gastrointestinal symptoms in patients on Elexacaftor/Tezacaftor/Ivacaftor

Journal of Pediatric Gastroenterology and Nutrition, Volume 82, Issue 1, Page 24-32, January 2026.
Abstract Objectives Multiorgan abdominal involvement is a hallmark of Cystic fibrosis (CF). The CFAbd‐Score© is the first CF‐specific gastrointestinal patient reported outcome‐measure (PROM) developed following FDA‐guidelines. The PROM has proved to sensitively differentiate people with CF (pwCF) from healthy controls (HC).
Isabelle Sermet‐Gaudelus, Pauline Sadrieh, Anne Sophie Bonnel, Raphael Enaud, Nathalie Wizla, Jeanne Languepin, Ambre Nangui, Audrey Deelawon, Johanna Rave, Samira Dabelow, Anton Barucha, Carlos Zagoya, Franziska Duckstein, Jochen Georg Mainz   +13 more
wiley   +1 more source

Capitalizing on the heterogeneous effects of CFTR nonsense and frameshift variants to inform therapeutic strategy for cystic fibrosis.

PLoS Genetics, 2018
CFTR modulators have revolutionized the treatment of individuals with cystic fibrosis (CF) by improving the function of existing protein. Unfortunately, almost half of the disease-causing variants in CFTR are predicted to introduce premature termination ...
Neeraj Sharma, Taylor A Evans, Matthew J Pellicore, Emily Davis, Melis A Aksit, Allison F McCague, Anya T Joynt, Zhongzhu Lu, Sangwoo T Han, Arianna F Anzmann, Anh-Thu N Lam, Abigail Thaxton, Natalie West, Christian Merlo, Laura B Gottschalk, Karen S Raraigh, Patrick R Sosnay, Calvin U Cotton, Garry R Cutting   +18 more
doaj   +1 more source

302 Clinical response to modulator therapy in patients homozygous for G27V CFTR

, 2023
J. Brewington, R. O’Shaughnessy, Kelly Thornton, A. Ostmann, J. Meeker, H. V. Morgan, Laura L. Walkup, Matthew M. Willmering, Jason C. Woods, Gary L. McPhail   +9 more
openalex   +1 more source

Mastering Organoid Growth: A Complete Guide to Overcoming Methodological Challenges

MedComm, Volume 7, Issue 1, January 2026.
Organoids offer transformative 3D models that precisely mimic human organ architecture and function. This review details the latest methodology, emphasizing the cultivation of liver and other abdominal organoids. We analyze technological challenges (e.g., nutrient delivery, scalability, diversity) and provide practical insights for new researchers ...
Chunbao Jiao, Omer Faruk Karakaya, Neda Dadgar, Chase J. Wehrle, Zahra Massoud, Hanna Hong, Robert L. Fairchild, Nic Leipzig, Federico Aucejo, Wen Wee Ma, Jan Joseph Melenhorst, Sofia Ferreira Gonzales, Andrea Schlegel   +12 more
wiley   +1 more source

326 Modulator response of rare CFTR missense variants not approved for elexacaftor-tezacaftor-ivacaftor

, 2023
Deepak Joshi, András Rab, Eric J. Sorscher, Jeong S. Hong   +3 more
openalex   +1 more source

Organoids in Cancer Research and Regenerative Medicine: Current Status, Challenges, and Future Prospects

MedComm, Volume 7, Issue 1, January 2026.
The management of cancer and tissue regeneration remains a substantial challenge to global health. Organoids, which are stem cell‐derived three‐dimensional multicellular structures, are revolutionizing biomedical research. Therefore, this review highlights their applications in cancer research and regenerative medicine, while discussing current ...
Ruiyang Li, Yuezhou Wu, Zhu'anzhen Zheng, Fengjin Zhou, Ke Xu, Jiacan Su   +5 more
wiley   +1 more source

Precision Medicine Based on CFTR Genotype for People with Cystic Fibrosis

Pharmacogenomics and Personalized Medicine, 2022
Iram Haq,1,2 Maryam Almulhem,1 Simone Soars,1 David Poulton,2,3 Malcolm Brodlie1,2 1Translational and Clinical Research Institute, Faculty of Medical Sciences, Newcastle University, Newcastle upon Tyne, UK; 2Paediatric Respiratory Medicine, Great North ...
Haq I, Almulhem M, Soars S, Poulton D, Brodlie M   +4 more
doaj  

Clinically approved CFTR modulators rescue Nrf2 dysfunction in cystic fibrosis airway epithelia [PDF]

, 2019
Dana C. Borcherding, Matthew Siefert, Songbai Lin, John J. Brewington, Hesham A. Sadek, John P. Clancy, Scott M. Plafker, Assem Ziady   +7 more
openalex   +1 more source