Results 41 to 50 of about 9,635 (207)

Bioengineered 3D hPSC‐Cholangiocyte Ducts With Physiological Signals for Biliary Disease Modeling

Advanced Healthcare Materials, EarlyView.
Tian and colleagues generated a bioengineered bile duct from human pluripotent stem cell (hPSC)‐derived intrahepatic cholangiocytes within a high‐throughput, 384‐well platform to systematically examine the influence of biliary physiological signals including fluid flow, stromal cells and bile acids, and models intrahepatic biliary disease progression ...
Britney Tian, Mina Ogawa, Manami Kondo, Gabrielle Langeveld, Ling Jun Huan, Feng Zhang, Andrew Hollinger, Sara Deir, Christine Bear, Boyang Zhang, Shinichiro Ogawa   +10 more
wiley   +1 more source

Cystic fibrosis modulator therapy can reverse cystic bronchiectasis

Respirology Case Reports, 2023
Bronchiectasis is often considered progressive and irreversible, so cases of regression or reversal are an important step in understanding the underlying pathophysiological mechanisms.
Peter G. Middleton, Nicholas J. Simmonds
doaj   +1 more source

Elexacaftor-Tezacaftor-Ivacaftor and Ivacaftor (Trikafta)

Canadian Journal of Health Technologies, 2021
CADTH reimbursement reviews are comprehensive assessments of the clinical effectiveness and cost-effectiveness, as well as patient and clinician perspectives, of a drug or drug class. The assessments inform non-binding recommendations that help guide the reimbursement decisions of Canada's federal, provincial, and territorial governments ...
openaire   +3 more sources

Integrating Human Intestinal Organoids into FDA'S New Approach Methodologies for Drug Discovery

Advanced Science, EarlyView.
Illustration summarizes how human intestinal organoids (HIOs) are becoming transformative in preclinical research. Preclinical drug discovery pipelines often rely on animal models for ADMET studies, even though interspecies ADME gaps, poor external validity, and high attrition rates are common.
Debarun Patra, Ibrahim M. Sayed, Souhrid Mukherjee, Honit Piplani, Aida Habtezion, Michael J. Rosen, Jospeh C. Wu   +6 more
wiley   +1 more source

Ivacaftor in cystic fibrosis adults: Czech experience with six years of follow-up

Biomedical Papers, 2016
Aims: Ivacaftor is a revolutionary treatment option for cystic fibrosis (CF) patients with G551D and other gating mutations. The aim of this study was to evaluate the clinical status of patients on ivacaftor who were followed for up to 6 years together ...
Libor Fila, Lucie Valentova Bartakova, Alzbeta Grandcourtova, Miloslav Marel, Radovan Drnek, Alena Bilkova, Milan Macek, Pavel Drevinek   +7 more
doaj   +1 more source

Nanomolar-potency 'co-potentiator' therapy for cystic fibrosis caused by a defined subset of minimal function CFTR mutants. [PDF]

, 2019
Available CFTR modulators provide no therapeutic benefit for cystic fibrosis (CF) caused by many loss-of-function mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) chloride channel, including N1303K. We previously introduced the
Finkbeiner, Walter E, Haggie, Peter M, Nielson, Dennis W, Phuan, Puay-Wah, Rivera, Amber A, Tan, Joseph-Anthony, Verkman, Alan S, Zlock, Lorna   +7 more
core  

A CFTR Potentiator in Patients with Cystic Fibrosis and the G551D Mutation [PDF]

, 2011
BACKGROUND: Increasing the activity of defective cystic fibrosis transmembrane conductance regulator (CFTR) protein is a potential treatment for cystic fibrosis.
Bell, Scott C., Davies, Jane, Dong, Qunming, Drevinek, Pavel, Elborn, J. Stuart, Griese, Matthias, Konstan, Michael W., McElvaney, N. Gerard, McKone, Edward F., Moss, Richard, Ordonez, Claudia, Ramsey, Bonnie W., Ratjen, Felix, Rodriguez, Sally, Rowe, Steven M., Sermet-Gaudelus, Isabelle, Tullis, Elizabeth, Wainwright, Claire E., Yen, Karl   +18 more
core   +1 more source

Uncovering Cystic Fibrosis Carrier: Insights From a Heterozygous CFTR‐F508del Rabbit Model

International Forum of Allergy &Rhinology, EarlyView.
ABSTRACT Background Chronic rhinosinusitis (CRS) is a heterogeneous inflammatory disorder frequently associated with impaired mucociliary clearance and bacterial infection. Individuals carrying a single cystic fibrosis transmembrane conductance regulator (CFTR) mutation exhibit partial CFTR dysfunction and are increasingly recognized as being at risk ...
Do‐Yeon Cho, Alexis E. McFeely, Daniel Skinner, Shaoyan Zhang, Justin Hedvat, Weslee Ping‐ay, Daniel M. Beswick, Justin H. Turner, Bradford A. Woodworth, Jie Xu   +9 more
wiley   +1 more source

Strategies against nonsense: oxadiazoles as translational readthrough-inducing drugs (TRIDs) [PDF]

, 2019
This review focuses on the use of oxadiazoles as translational readthrough-inducing drugs (TRIDs) to rescue the functional full-length protein expression in mendelian genetic diseases caused by nonsense mutations.
Campofelice A., Di Leonardo A., Lentini L., Melfi R., Pace A., Pibiri I., Tutone M.   +6 more
core   +1 more source

Determining the Minimal Clinically Important Difference of the 40‐Item Smell Identification Test in People With Cystic Fibrosis

International Forum of Allergy &Rhinology, EarlyView.
ABSTRACT Background Chronic rhinosinusitis (CRS) and olfactory dysfunction (OD) are highly prevalent among people with cystic fibrosis (PwCF) and negatively impact quality of life. The 40‐item Smell Identification Test (SIT) is widely used to assess psychophysical olfaction, but a CF‐specific minimal clinically important difference (MCID) has not been ...
Eugene Oh, Jessa E. Miller, Michelle J. Lee, Anna Zemke, David Baraghoshi, Matthew J. Strand, Jeremiah A. Alt, Todd E. Bodner, Michael Chang, Naweed I. Chowdhury, Patricia H. Eshaghian, Anne E. Getz, Jennifer L. Goralski, David A. Gudis, Peter H. Hwang, Ashoke Khanwalkar, Adam J. Kimple, Jivianne T. Lee, Douglas A. Li, Jess C. Mace, Jayakar V. Nayak, Jonathan B. Overdevest, Zara Patel, Daniella K. Safatian, Rodney J. Schlosser, Brent Senior, Amanda L. Stapleton, Timothy L. Smith, Zachary M. Soler, Jeffrey D. Suh, Grant A. Turner, Marilene B. Wang, Milene T. Saavedra, Jennifer L. Taylor‐Cousar, Daniel M. Beswick   +34 more
wiley   +1 more source