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Biomaterials-mediated CRISPR/Cas9 delivery: recent challenges and opportunities in gene therapy
The use of biomaterials in delivering CRISPR/Cas9 for gene therapy in infectious diseases
holds tremendous potential. This innovative approach combines the advantages of …
holds tremendous potential. This innovative approach combines the advantages of …
A new era of targeting cystic fibrosis with non-viral delivery of genomic medicines
Cystic fibrosis (CF) is a complex genetic respiratory disorder that necessitates innovative
gene delivery strategies to address the mutations in the gene. This review delves into the …
gene delivery strategies to address the mutations in the gene. This review delves into the …
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 …
Enhancing in vivo cell and tissue targeting by modulation of polymer nanoparticles and macrophage decoys
The in vivo efficacy of polymeric nanoparticles (NPs) is dependent on their
pharmacokinetics, including time in circulation and tissue tropism. Here we explore the …
pharmacokinetics, including time in circulation and tissue tropism. Here we explore the …
Cystic fibrosis and fat malabsorption: Pathophysiology of the cystic fibrosis gastrointestinal tract and the impact of highly effective CFTR modulator therapy
CM McDonald, EK Reid, JF Pohl… - Nutrition in Clinical …, 2024 - Wiley Online Library
Cystic fibrosis (CF) is a progressive, genetic, multi‐organ disease affecting the respiratory,
digestive, endocrine, and reproductive systems. CF can affect any aspect of the …
digestive, endocrine, and reproductive systems. CF can affect any aspect of the …
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 …
A red light-triggered chemical tool for sequence-specific alkylation of G-quadruplex and I-motif DNA
E Cadoni, L De Paepe, G Colpaert, R Tack… - Nucleic Acids …, 2023 - academic.oup.com
The importance of non-canonical DNA structures such as G-quadruplexes (G4) and
intercalating-motifs (iMs) in the fine regulation of a variety of cellular processes has been …
intercalating-motifs (iMs) in the fine regulation of a variety of cellular processes has been …
Recent progress in nanomedicine-mediated cytosolic delivery
H Son, J Shin, J Park - RSC advances, 2023 - pubs.rsc.org
Cytosolic delivery of bioactive agents has exhibited great potential to cure undruggable
targets and diseases. Because biological cell membranes are a natural barrier for living …
targets and diseases. Because biological cell membranes are a natural barrier for living …
Ligand-free biodegradable poly (beta-amino ester) nanoparticles for targeted systemic delivery of mRNA to the lungs
Non-viral nanoparticles (NPs) have seen heightened interest as a delivery method for a
variety of clinically relevant nucleic acid cargoes in recent years. While much of the focus …
variety of clinically relevant nucleic acid cargoes in recent years. While much of the focus …
[HTML][HTML] Recent advancements in development and therapeutic applications of genome-targeting triplex-forming oligonucleotides and peptide nucleic acids
Y Mikame, A Yamayoshi - Pharmaceutics, 2023 - mdpi.com
Recent developments in artificial nucleic acid and drug delivery systems present
possibilities for the symbiotic engineering of therapeutic oligonucleotides, such as antisense …
possibilities for the symbiotic engineering of therapeutic oligonucleotides, such as antisense …