Results 91 to 100 of about 71,270 (242)
ABSTRACT Lipid nanoparticles (LNPs) represent the most clinically advanced platform for RNA delivery and have enabled major breakthroughs in vaccines and gene therapies. However, their broader application is still limited by inefficient extrahepatic delivery, immunogenicity, and insufficient control over tissue‐ and cell‐specific targeting. This review
Yu Han +5 more
wiley +1 more source
Insulin-like growth factor 1 (IGF-1) enhances the protein expression of CFTR. [PDF]
Low levels of insulin-like growth factor 1 (IGF-1) have been observed in the serum of cystic fibrosis (CF) patients. However, the effects of low serum IGF-1 on the cystic fibrosis transmembrane conductance regulator (CFTR), whose defective function is ...
Ha Won Lee +4 more
doaj +1 more source
Two Small Molecules Restore Stability to a Sub-population of the Cystic Fibrosis Transmembrane conductance Regulator with the Predominant Disease-causing Mutation [PDF]
Cystic fibrosis (CF) is caused by mutations that disrupt the plasma membraneexpression, stability, and function of the cystic fibrosis transmembrane conductance regulator (CFTR) Cl- channel.
Cai, Zhiwei +8 more
core +2 more sources
Abstract Objective To summarize the electro‐clinical and genetic characteristics of children with Mowat–Wilson syndrome (MWS). Methods This study is a hospital‐based case series analyzing clinical data from 31 pediatric patients with MWS and epilepsy treated at Peking University First Hospital between June 2020 and December 2024.
Yi Ju, Tao‐yun Ji
wiley +1 more source
Long term in vitro expansion of epithelial stem cells enabled by pharmacological inhibition of PAK1-ROCK-Myosin II and TGF-β signaling [PDF]
Summary: Despite substantial self-renewal capability in vivo, epithelial stem and progenitor cells located in various tissues expand for a few passages in vitro in feeder-free condition before they succumb to growth arrest.
Challberg, Sharon S +7 more
core +2 more sources
Impact of organic pollutants on phenotype and gene expression in human breast cancer cells
ABSTRACT Human exposure to industrial chemical compounds is widespread and, although often beneficial, prolonged contact may contribute to disease development, including cancer. While many studies have shown organic pollutants (OP) are cytotoxic, few have explored how long‐term exposure alters cell phenotype.
Camila Confortin +7 more
wiley +1 more source
Setting priorities for development of emerging interventions against childhood diarrhoea [PDF]
An expert panel exercise was conducted to assess feasibility and potential effectiveness of 10 emerging health interventions against childhood diarrhoea. Twelve international experts were invited to take part in a CHNRI priority setting process.
Bhutta +18 more
core +1 more source
Abstract Infantile exocrine pancreatic insufficiency is a rare condition, most often encountered in the context of cystic fibrosis or Shwachman–Diamond syndrome. The SPINK1 gene encodes a trypsin inhibitor protein that prevents the premature activation of digestive enzymes in pancreatic tissue.
France Chalon +10 more
wiley +1 more source
Elexacaftor–Tezacaftor–Ivacaftor Reduces Revision Sinus Surgery in People With Cystic Fibrosis
Elexacaftor–tezacaftor–ivacaftor (ETI) was associated with a significant decrease in the frequency and rate of endoscopic sinus surgeries in patients with cystic fibrosis. These findings suggest an improvement in CF‐related chronic rhinosinusitis outcomes following ETI and may influence future CF treatment decisions.
Amy Lin +6 more
wiley +1 more source

