Results 281 to 290 of about 1,132,527 (387)

A CFTR potentiator in patients with cystic fibrosis and the G551D mutation.

New England Journal of Medicine, 2011
N. England, M. Bonnie W. Ramsey, M. Jane Davies, N. G. M. Ch.B., Elizabeth Tullis M.D., M. S. C. Bell, Pavel D ř evínek M.D., Matthias Griese M.D., Edward F. McKone M.D., M. C. E. Wainwright, Michael W. Konstan B.S., Richard Moss M.D., Felix Ratjen M.D., Ph. Isabelle Sermet-Gaudelus, Steven M. Rowe Ph.D., Qunming Dong M.S.P.H., Sally Rodriguez Ph.D., M. K. Yen, Claudia Ordoñez M.D., M. J. S. Elborn   +19 more
semanticscholar   +1 more source

Nature Inspired Delivery Vehicles for CRISPR‐Based Genome Editing

Small, EarlyView.
The review highlights nature‐inspired nanocarriers for CRISPR delivery, emphasizing viral vectors, extracellular vesicles, liposomes, and lipid nanoparticles. It discusses their roles in improving specificity, minimizing immunogenicity, and overcoming barriers in genome editing. Recent advancements, challenges, and therapeutic applications are explored,
Elizabeth Maria Clarissa, Mamata Karmacharya, Hyunmin Choi, Sumit Kumar, Yoon‐Kyoung Cho   +4 more
wiley   +1 more source

Evaluating Georgia's Cystic Fibrosis Newborn Screening Algorithm to Inform Improvement Recommendations. [PDF]

Int J Neonatal Screen
Truitt B, Barr E, Wittenauer A, Jergel A, Bai S, Sanchez Russo R, Oliver KE, McKie K, Linnemann RW.   +8 more
europepmc   +1 more source

Restoring Cystic Fibrosis Transmembrane Conductance Regulator Function Reduces Airway Bacteria and Inflammation in People with Cystic Fibrosis and Chronic Lung Infections

American Journal of Respiratory and Critical Care Medicine, 2017
K. B. Hisert, S. Heltshe, C. Pope, P. Jorth, Xia Wu, R. M. Edwards, M. Radey, F. Accurso, D. Wolter, G. Cooke, Ryan J Adam, S. Carter, B. Grogan, J. Launspach, S. Donnelly, C. Gallagher, J. Bruce, D. Stoltz, M. Welsh, L. Hoffman, E. McKone, Pradeep K. Singh   +21 more
semanticscholar   +1 more source

Organoid‐based novel technology for antitumor drug screening

VIEW, EarlyView.
Organoids represent a cutting‐edge research model that closely mimics in vivo organ physiology, making them highly valuable for tumor immunotherapy drug screening. This review explores the integration of organoids with advanced technologies, such as gene editing, 3D bioprinting, and AI, highlighting novel fusion platforms and their applications.
Yu Su, Yuan Chen, Guanning Wei, Yihai Shi   +3 more
wiley   +1 more source

Sinonasal Disease in Pediatric Patients With Cystic Fibrosis and the Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) I1234V Mutation in Qatar. [PDF]

Cureus
Odeh M, Al-Naimi AR, Younes A.
europepmc   +1 more source

Clinical and radiographic features of a family with hypochondroplasia owing to a novel Asn540Ser mutation in the fibroblast growth factor receptor 3 gene [PDF]

, 2000
Craen, Margarita, De Paepe, Anne, Leroy, Juliaan, Mortier, Geert, Nuytinck, Lieve, Renard, Jean-Pierre   +5 more
core   +3 more sources

State-of-the-Art Review: Transformative Changes in the Care of People With Cystic Fibrosis: Implications for Infectious Diseases Specialists [PDF]

Lisa Saiman, Laurie Murray, Anne Brown
openalex   +1 more source

Pseudomonas aeruginosa Evolutionary Adaptation and Diversification in Cystic Fibrosis Chronic Lung Infections

Trends in Microbiology, 2016
C. Winstanley, Siobhán O’Brien, M. Brockhurst   +2 more
semanticscholar   +1 more source

Chinese Guidelines for Diagnosis and Treatment of Chronic Rhinosinusitis (2024)

World Journal of Otorhinolaryngology - Head and Neck Surgery, EarlyView.
ABSTRACT Chronic rhinosinusitis (CRS), a complex inflammatory disease with heterogeneous pathogenesis, demands evolving evidence‐based strategies. Since the 2018 Chinese guidelines and EPOS2020, international advances in CRS immunopathology and biologics have revolutionized therapeutic approaches, particularly through phenotype–endotype classification ...
Subspecialty Group of Rhinology, Editorial Board of Chinese Journal of Otorhinolaryngology Head and Neck Surgery, Subspecialty Group of Rhinology, Society of Otorhinolaryngology Head and Neck Surgery, Chinese Medical Association   +4 more
wiley   +1 more source