Results 191 to 200 of about 1,231,622 (330)

Lipid Nanoparticles for Delivery of CRISPR Gene Editing Components

Small Methods, Volume 10, Issue 2, 22 January 2026.
The review presents a comprehensive overview of each component of lipid nanoparticles（LNPs）and their effects on editing efficiency. It specifically highlights strategies for achieving non‐liver delivery, aiming for broader applications in gene editing. Furthermore, this review summarizes the applications of LNPs in gene editing and offers insights for ...
Fan Wu, Nei Li, Yudian Xiao, Rohan Palanki, Hannah Yamagata, Michael J. Mitchell, Xuexiang Han   +6 more
wiley   +1 more source

Adenine base editing of CFTR using receptor targeted nanoparticles restores function to G542X cystic fibrosis airway epithelial cells

Cellular and Molecular Life Sciences
The cystic fibrosis (CF) causing variant G542X harbours a premature translation stop signal in the cystic fibrosis transmembrane conductance regulator (CFTR) mRNA. This results in nonsense-mediated decay and loss of functional CFTR protein which leads to
Isabelle Rose, Miriam Greenwood, Matthew Biggart, Natalie Baumlin, Robert Tarran, Stephen L. Hart, Deborah L. Baines   +6 more
doaj   +1 more source

β‐Hydroxybutyrate Acts as an Exercise Mimetic to Protect the Aging Liver

Aging Cell, Volume 25, Issue 1, January 2026.
β‐hydroxybutyrate (β‐HB) acts as an exercise mimetic to protect the aging liver. In aged models, exercise‐induced β‐HB delays liver aging by reversing macrophage senescence, blocking mtDNA/cGAS‐STING‐driven inflammation, and alleviating hepatocyte lipid deposition/PANoptosis.
Ke Li, Lian Wang, Danlin Zhu, Wenhong Wang, Yifan Guo, Haoyang Gao, Muge Zhou, Weihua Xiao   +7 more
wiley   +1 more source

Lumacaftor-ivacaftor in the treatment of cystic fibrosis: design, development and place in therapy

Drug Design, Development and Therapy, 2019
GJ ConnettNational Institute for Health Research, Southampton Respiratory Biomedical Research Centre, University Hospital Southampton NHS Foundation Trust, Southampton, SO16 6YD, UKAbstract: Lumacaftor-ivacaftor is a combination of two small molecule ...
Connett GJ
doaj  

Unity Is Strength: The Mutual Alliance between CFTR and SLC26A6 as Therapeutic Opportunity in Cystic Fibrosis

Pharmaceuticals
Patients with cystic fibrosis (PwCF) have recently experienced an unprecedented breakthrough with the adoption of modulator therapy in clinical practice.
Marilena Pariano, Cinzia Antognelli, Luigina Romani, Claudio Costantini   +3 more
doaj   +1 more source

311 Active Pseudomonas aeruginosa infection of primary airway epithelia enhances CFTR modulator rescue [PDF]

, 2023
Deborah M. Cholon, M. Higgs, Susan E. Boyles, Nancy L. Quinney, S. Meinig, John T. Minges, Carla M. P. Ribeiro, Matthew C. Wolfgang, Martina Gentzsch   +8 more
openalex   +1 more source

Novel CFTR Modulators for Treating Cystic Fibrosis [PDF]

ACS Medicinal Chemistry Letters, 2021
openaire   +3 more sources

UK Biobank: Transforming drug discovery and precision medicine

British Journal of Pharmacology, Volume 183, Issue 2, Page 234-248, January 2026.
UK Biobank is a large‐scale, prospective study with extensive genetic and phenotypic data on half a million individuals. Volunteers, aged between 40 and 69 years, were recruited between 2006 and 2010 from the general population of the United Kingdom.
Jelena Bešević, Saredo Said, Reka Nagy, Yalda Jamshidi, Christopher D. Whelan, Lauren Carson, Martin K. Rutter, Adam J. Lewandowski, Mark Effingham, Rory Collins, Ben Lacey, Naomi E. Allen   +11 more
wiley   +1 more source

Lumacaftor/ivacaftor in patients with cystic fibrosis and advanced lung disease homozygous for F508del-CFTR [PDF]

, 2018
Atkinson, Jeffrey, Barto, Tara Lynn, Haddad, Tarik, Jain, Manu, Marigowda, Gautham, Pilewski, Joseph, Tang, Rui, Taylor-Cousar, Jennifer L, Tian, Simon, VH14-809-106 Investigator Group,, Waltz, David   +10 more
core   +2 more sources

Cystic fibrosis-related liver disease: clinical presentations, diagnostic and monitoring approaches in the era of CFTR modulator therapies.

Journal of Hepatology, 2021
Jérémy Dana, D. Debray, A. Beaufrère, S. Hillaire, M. Fabre, C. Reinhold, T. Baumert, L. Berteloot, V. Vilgrain   +8 more
semanticscholar   +1 more source