[HTML][HTML] Gene therapy for cystic fibrosis: Challenges and prospects
H Sui, X Xu, Y Su, Z Gong, M Yao, X Liu… - Frontiers in …, 2022 - frontiersin.org
Cystic fibrosis (CF) is a life-threatening autosomal-recessive disease caused by mutations in
a single gene encoding cystic fibrosis transmembrane conductance regulator (CFTR). CF …
a single gene encoding cystic fibrosis transmembrane conductance regulator (CFTR). CF …
[HTML][HTML] Epithelial Transport in Disease: An Overview of Pathophysiology and Treatment
VJ Clemente-Suárez, A Martín-Rodríguez… - Cells, 2023 - mdpi.com
Epithelial transport is a multifaceted process crucial for maintaining normal physiological
functions in the human body. This comprehensive review delves into the pathophysiological …
functions in the human body. This comprehensive review delves into the pathophysiological …
[HTML][HTML] Bile acid-containing lipid nanoparticles enhance extrahepatic mRNA delivery
Lipid nanoparticles (LNPs) have emerged as a viable, clinically-validated platform for the
delivery of mRNA therapeutics. LNPs have been utilized as mRNA delivery systems for …
delivery of mRNA therapeutics. LNPs have been utilized as mRNA delivery systems for …
[HTML][HTML] Applications of human organoids in the personalized treatment for digestive diseases
Q Wang, F Guo, Y Jin, Y Ma - Signal Transduction and Targeted …, 2022 - nature.com
Digestive system diseases arise primarily through the interplay of genetic and environmental
influences; there is an urgent need in elucidating the pathogenic mechanisms of these …
influences; there is an urgent need in elucidating the pathogenic mechanisms of these …
Current knowledge on the tissue distribution of mRNA nanocarriers for therapeutic protein expression
Exogenously delivered mRNA-based drugs are emerging as a new class of therapeutics
with the potential to treat several diseases. Over the last decade, advancements in the …
with the potential to treat several diseases. Over the last decade, advancements in the …
[HTML][HTML] The Role of MMPs in the Era of CFTR Modulators: An Additional Target for Cystic Fibrosis Patients?
R Esposito, D Mirra, G Spaziano, F Panico, L Gallelli… - Biomolecules, 2023 - mdpi.com
Cystic fibrosis (CF) is a high-prevalence disease characterized by significant lung
remodeling, responsible for high morbidity and mortality worldwide. The lung structural …
remodeling, responsible for high morbidity and mortality worldwide. The lung structural …
[HTML][HTML] CFTR RNA-and DNA-based therapies
PT Harrison - Current Opinion in Pharmacology, 2022 - Elsevier
This review provides an update on recent developments of RNA-and DNA-based
methodologies and their intracellular targets in the context of cystic fibrosis (CF) lung …
methodologies and their intracellular targets in the context of cystic fibrosis (CF) lung …
Mucus-targeting therapies of defective mucus clearance for cystic fibrosis: a short review
MF Figueira, CMP Ribeiro, B Button - Current opinion in pharmacology, 2022 - Elsevier
In the lungs, defective CFTR associated with cystic fibrosis (CF) represents the nidus for
abnormal mucus clearance in the airways and consequently a progressive lung disease …
abnormal mucus clearance in the airways and consequently a progressive lung disease …
[HTML][HTML] CRISPR-Cas9 Direct Fusions for Improved Genome Editing via Enhanced Homologous Recombination
T Tabassum, G Pietrogrande, M Healy… - International Journal of …, 2023 - mdpi.com
DNA repair in mammalian cells involves the coordinated action of a range of complex
cellular repair machinery. Our understanding of these DNA repair processes has advanced …
cellular repair machinery. Our understanding of these DNA repair processes has advanced …
[HTML][HTML] Repeat or single-dose lentiviral vector administration to mouse lungs? It's all about the timing
M Donnelley, P Cmielewski, E Knight, C Carpentieri… - Gene Therapy, 2023 - nature.com
Lentiviral vectors are attractive delivery vehicles for cystic fibrosis gene therapy owing to
their low immunogenicity and ability to integrate into the host cell genome, thereby …
their low immunogenicity and ability to integrate into the host cell genome, thereby …