Results 101 to 110 of about 7,856 (243)

Prime editing in neuropsychiatric disorders: From mutation‐specific target selection to clinical translation

Neuroprotection, EarlyView.
Abstract Prime editing, a novel clustered regularly interspaced short palindromic repeats (CRISPR)‐based technology, fuses a reverse transcriptase (RT) to an engineered CRISPR‐associated protein 9 (Cas9) and uses a prime editing guide RNA (pegRNA)‐encoded template.
Tianshan Ji, Yuan Zhang, Jinyi Zhao, Yiping Lu, Chengkun Wang   +4 more
wiley   +1 more source

Adherence to cystic fibrosis transmembrane conductance regulator (CFTR) modulators: analysis of a national specialty pharmacy database

, 2021
Background: There have been significant advances in Cystic Fibrosis (CF) treatment, with the introduction of Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) modulators.
Mehta, Zumi, Covvey, Jordan R, Giannetti, Vincent, Kamal, Khalid M, Miller, Richard   +4 more
core   +1 more source

State-dependent modulation of CFTR gating by pyrophosphate [PDF]

Journal of General Physiology, 2009
Cystic fibrosis transmembrane conductance regulator (CFTR) is an adenosine triphosphate (ATP)-gated chloride channel. ATP-induced dimerization of CFTR's two nucleotide-binding domains (NBDs) has been shown to reflect the channel open state, whereas hydrolysis of ATP is associated with channel closure.
Tsai, Ming-Feng, Shimizu, Hiroyasu, Sohma, Yoshiro, Li, Min, Hwang, Tzyh-Chang   +4 more
openaire   +2 more sources

Current Cell/Organoid and Animal Models for Primary Sclerosing Cholangitis

Portal Hypertension &Cirrhosis, EarlyView.
Primary sclerosing cholangitis (PSC) is a chronic cholestatic liver disease with limited therapeutic options and a marked risk of progression to biliary fibrosis, cirrhosis, and malignancy. Progress in PSC research has been hindered by the lack of models that faithfully recapitulate the complex biliary microenvironment and disease heterogeneity ...
Qigu Yao, Sheng Cheng, Qiaoling Pan, Jiong Yu, Hongcui Cao   +4 more
wiley   +1 more source

Flavonoids in Nasal Therapeutics: Biological Activities, Delivery Challenges, and Formulation Strategies–A Systematic Review

Phytotherapy Research, EarlyView.
Overview of the therapeutic potential of flavonoids in nasal delivery systems, highlighting their benefits to the human nasal cavity, and applications in the treatment of respiratory and brain‐related diseases. ABSTRACT Flavonoids represent a significant group of secondary metabolites.
Jeniffer Viviana Ramirez Hernández, Sara Elis Bianchi, Flávia Nathiely Silveira Fachel, Elizandra Braganhol, Sheila Porto de Matos, Valquíria Linck Bassani   +5 more
wiley   +1 more source

Impact of Cystic Fibrosis Transmembrane Conductance Regulator Modulating Therapies on Liver Transplant Outcomes

Gastro Hep Advances
Background and Aims: Up to 40% of patients with cystic fibrosis (CF) develop CF-related liver disease (CFrLD), which can progress to the point of requiring liver transplantation (LT).
Sara Naimimohasses, Ankit Ray, Eunice Tan, Asher Wiggins, Bima J. Hasjim, Shiyi Chen, Mamatha Bhat   +6 more
doaj   +1 more source

Update on Non‐Biological and RNA‐Based Therapeutics in Chronic Inflammatory Diseases: Precision Medicine Through Small Molecules: An EAACI Position Paper

Allergy, EarlyView.
ABSTRACT In the last decades, critical advancements in research technology and knowledge on disease mechanisms steered therapeutic approaches for chronic inflammatory diseases towards unprecedented target specificity. For allergic and chronic lung diseases, biologic drugs pioneered this goal, acquiring on the way—through the clinical use of monoclonal ...
F. Roth‐Walter, I. M. Adcock, C. Benito‐Villalvilla, R. Bianchini, L. Bjermer, G. Caramori, L. Cari, K. F. Chung, Z. Diamant, I. Eguiluz‐Gracia, E. F. Knol, M. Jesenak, F. Levi‐Schaffer, G. Nocentini, L. O'Mahony, O. Palomares, F. Redegeld, M. Sokolowska, B. Van Esch, M. Zurlo, C. Stellato   +20 more
wiley   +1 more source

Triplet CFTR modulators: future prospects for treatment of cystic fibrosis

, 2018
Nauman Chaudary Division of Pulmonary Disease and Critical Care Medicine, Department of Medicine, Virginia Commonwealth University, Richmond, VA, USA Abstract: Cystic fibrosis (CF) is an autosomal recessive genetic disease characterized by mutations in
Chaudary N
core  

Airway Inflammation and Host Responses in the Era of CFTR Modulators

, 2020
The arrival of cystic fibrosis transmembrane conductance regulator (CFTR) modulators as a new class of treatment for cystic fibrosis (CF) in 2012 represented a pivotal advance in disease management, as these small molecules directly target the upstream ...
Daniel F. McAuley, Creane, Shannice, Taggart, Clifford C, Sinéad Weldon, Declan F. Doherty, Claire Houston, Joseph C. Kidney, Michael C. McKelvey, McKelvey, Michael C, McAuley, Daniel F; id_orcid, Karen Keown, Clifford C. Taggart, Downey, Damian G; id_orcid, Brown, Ryan, Damian G. Downey, Kidney, Joseph C, Dermot Linden, Doherty, Declan F, Shannice Creane, Houston, Claire, Keown, Karen; id_orcid, Linden, Dermot, Ryan Brown, Weldon, Sinéad   +23 more
core   +1 more source

Differential Roles for IL‐4Rα and IL‐13Rα1 in Immune Cell Infiltration and Epithelial Remodeling in Experimental Eosinophilic Gastritis

Allergy, EarlyView.
This study described the development of an experimental model for eosinophilic gastritis (EoG). Experimental EoG recapitulates histopathological features of human disease. including foveolar elongation, fibrosis, eosinophilia, mastocytosis and shared transcriptional programs.
Anish Dsilva, Shraddha Sharma, Shireen Barakey, Ariel Wagner, Alice Keisar, Tali Bar‐on, Michal Itan, Ariel Munitz   +7 more
wiley   +1 more source