Results 91 to 100 of about 1,459,532 (261)
, 2023 The presence of pathogenic variants in the CFTR gene causes cystic fibrosis (CF) through various molecular mechanisms that affect the formation and functional activity of the CFTR chloride channel.
N. V. Bulatenko +13 more
core +1 more source
Peripheral Protein Quality Control as a Novel Drug Target for CFTR Stabilizer
Frontiers in Pharmacology, 2018 Conformationally defective cystic fibrosis transmembrane conductance regulator (CFTR) including rescued ΔF508-CFTR is rapidly eliminated from the plasma membrane (PM) even in the presence of a CFTR corrector and potentiator, limiting the therapeutic ...
Ryosuke Fukuda, Tsukasa Okiyoneda
doaj +1 more source
BMEMat, EarlyView.The effects of NETs on regeneration of various diabetic tissues, and strategies targeting NETs for diabetes tissue regeneration. In the diabetic environment, NETs undergo complex metabolic and immune reprogramming, leading to dynamic changes in antibacterial and proinflammatory functions, and affecting regeneration of multiple systemic tissues.
Xinyi Jiang +6 more
wiley +1 more source
Changes in fecal microbiota with CFTR modulator therapy: A pilot study
, 2021 Studies have demonstrated that people with CF with pancreatic insufficiency (PI) have fecal dysbioses. Evidence suggests the cause of these dysbioses to be multifactorial, and that important drivers include antibiotic exposure, dietary intake, and CF ...
Singh, PK +31 more
core +1 more source
Scientific Reports, 2020 Cystic fibrosis (CF) is characterized by chronic bacterial infections and heightened inflammation. Widespread ineffective antibiotic use has led to increased isolation of drug resistant bacterial strains from respiratory samples.
Chandra L. Shrestha +6 more
doaj +1 more source
Status and future of recombinant adeno‐associated virus vector manufacturing
Biotechnology Progress, EarlyView.Abstract Sixty years of adeno‐associated virus (AAV) research illustrates a trajectory marked by basic science exploration, iterative innovation, persistent challenges, a number of clinical setbacks, as well as commercial therapeutic triumphs. This continual evolution has led to recombinant AAV (rAAV) becoming a cornerstone of modern gene therapy ...
Frank Agbogbo, David Dismuke
wiley +1 more source
Setting the stage for CFTR modulator studies in infants [PDF]
Thorax, 2014 Because irreversible damage to the cystic fibrosis (CF) lung is already observed in infancy, a need has arisen to assess and monitor lung function early in life. Infant lung function testing (ILFT) is non-invasive, does not require radiation or anaesthesia and can be repeated frequently; therefore, development, standardisation and validation of ...
openaire +3 more sources
JPGN Reports, EarlyView.Abstract Objectives The efficacy of cystic fibrosis transmembrane conductance regulator (CFTR)‐modulator therapies in preventing or ameliorating cystic fibrosis liver disease (CFLD) by correcting CFTR in cholangiocytes is not well‐documented. This study aimed to assess liver function during CFTR‐modulators.
Laura Giugliano +12 more
wiley +1 more source
Impact of CFTR modulator use on outcomes in people with severe cystic fibrosis lung disease
European Respiratory Review, 2020 Drug compounds that augment the production and activity of the cystic fibrosis (CF) transmembrane regulator (CFTR) have revolutionised CF care. Many adults and some children with CF suffer advanced and severe lung disease or await lung transplantation ...
M. Shteinberg, J. Taylor-Cousar
semanticscholar +1 more source
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 +4 more
wiley +1 more source