Biomaterials-mediated CRISPR/Cas9 delivery: recent challenges and opportunities in gene therapy

AK Dubey, E Mostafavi - Frontiers in Chemistry, 2023 - frontiersin.org
The use of biomaterials in delivering CRISPR/Cas9 for gene therapy in infectious diseases
holds tremendous potential. This innovative approach combines the advantages of …

A new era of targeting cystic fibrosis with non-viral delivery of genomic medicines

NTV Murthy, K Vlasova, J Renner, A Jozic… - Advanced drug delivery …, 2024 - Elsevier
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 …

Lung SORT LNPs enable precise homology-directed repair mediated CRISPR/Cas genome correction in cystic fibrosis models

T Wei, Y Sun, Q Cheng, S Chatterjee, Z Traylor… - Nature …, 2023 - nature.com
Abstract Approximately 10% of Cystic Fibrosis (CF) patients, particularly those with CF
transmembrane conductance regulator (CFTR) gene nonsense mutations, lack effective …

Enhancing in vivo cell and tissue targeting by modulation of polymer nanoparticles and macrophage decoys

AS Piotrowski-Daspit, LG Bracaglia, DA Eaton… - Nature …, 2024 - nature.com
The in vivo efficacy of polymeric nanoparticles (NPs) is dependent on their
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 …

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 …

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 …

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 …

Ligand-free biodegradable poly (beta-amino ester) nanoparticles for targeted systemic delivery of mRNA to the lungs

EW Kavanagh, SY Tzeng, N Sharma, GR Cutting… - Biomaterials, 2025 - Elsevier
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 …

[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 …