[HTML][HTML] Future therapies for cystic fibrosis
We are currently witnessing transformative change for people with cystic fibrosis with the
introduction of small molecule, mutation-specific drugs capable of restoring function of the …
introduction of small molecule, mutation-specific drugs capable of restoring function of the …
[HTML][HTML] Gene therapy for cystic fibrosis: new tools for precision medicine
The discovery of the Cystic fibrosis (CF) gene in 1989 has paved the way for incredible
progress in treating the disease such that the mean survival age of individuals living with CF …
progress in treating the disease such that the mean survival age of individuals living with CF …
Engineering lipid nanoparticles for enhanced intracellular delivery of mRNA through inhalation
Despite lipid nanoparticles'(LNPs) success in the effective and safe delivery of mRNA
vaccines, an inhalation-based mRNA therapy for lung diseases remains challenging. LNPs …
vaccines, an inhalation-based mRNA therapy for lung diseases remains challenging. LNPs …
[HTML][HTML] Lung SORT LNPs enable precise homology-directed repair mediated CRISPR/Cas genome correction in cystic fibrosis models
Abstract Approximately 10% of Cystic Fibrosis (CF) patients, particularly those with CF
transmembrane conductance regulator (CFTR) gene nonsense mutations, lack effective …
transmembrane conductance regulator (CFTR) gene nonsense mutations, lack effective …
[HTML][HTML] Induced Pluripotent Stem Cells in Disease Biology and the Evidence for Their In Vitro Utility
A Adegunsoye, NM Gonzales… - Annual Review of …, 2023 - annualreviews.org
Many human phenotypes are impossible to recapitulate in model organisms or immortalized
human cell lines. Induced pluripotent stem cells (iPSCs) offer a way to study disease …
human cell lines. Induced pluripotent stem cells (iPSCs) offer a way to study disease …
[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 …
Functional correction of CFTR mutations in human airway epithelial cells using adenine base editors
S Krishnamurthy, S Traore, AL Cooney… - Nucleic acids …, 2021 - academic.oup.com
Mutations in the CFTR gene that lead to premature stop codons or splicing defects cause
cystic fibrosis (CF) and are not amenable to treatment by small-molecule modulators. Here …
cystic fibrosis (CF) and are not amenable to treatment by small-molecule modulators. Here …
[HTML][HTML] Functional restoration of a CFTR splicing mutation through RNA delivery of CRISPR adenine base editor
S Amistadi, G Maule, M Ciciani, MM Ensinck… - Molecular Therapy, 2023 - cell.com
Cystic fibrosis (CF) is a genetic disease caused by mutations in the CF transmembrane
conductance regulator (CFTR) gene. The 2789+ 5G> A CFTR mutation is a quite frequent …
conductance regulator (CFTR) gene. The 2789+ 5G> A CFTR mutation is a quite frequent …
[HTML][HTML] Genome editing for cystic fibrosis
G Wang - Cells, 2023 - mdpi.com
Cystic fibrosis (CF) is a monogenic recessive genetic disorder caused by mutations in the
CF Transmembrane-conductance Regulator gene (CFTR). Remarkable progress in basic …
CF Transmembrane-conductance Regulator gene (CFTR). Remarkable progress in basic …
[HTML][HTML] Targeted replacement of full-length CFTR in human airway stem cells by CRISPR-Cas9 for pan-mutation correction in the endogenous locus
Cystic fibrosis (CF) is a monogenic disease caused by impaired production and/or function
of the CF transmembrane conductance regulator (CFTR) protein. Although we have …
of the CF transmembrane conductance regulator (CFTR) protein. Although we have …