Development of novel therapeutic agents by inhibition of oncogenic microRNAs

DD Nguyen, S Chang - International journal of molecular sciences, 2017 - mdpi.com
MicroRNAs (miRs, miRNAs) are regulatory small noncoding RNAs, with their roles already
confirmed to be important for post-transcriptional regulation of gene expression affecting cell …

Hydrogel-coated microneedle arrays for minimally invasive sampling and sensing of specific circulating nucleic acids from skin interstitial fluid

D Al Sulaiman, JYH Chang, NR Bennett, H Topouzi… - ACS …, 2019 - ACS Publications
Minimally invasive technologies that can sample and detect cell-free nucleic acid
biomarkers from liquid biopsies have recently emerged as clinically useful for early …

[HTML][HTML] One size does not fit all: the past, present and future of cystic fibrosis causal therapies

MM Ensinck, MS Carlon - Cells, 2022 - mdpi.com
Cystic fibrosis (CF) is the most common monogenic disorder, caused by mutations in the CF
transmembrane conductance regulator (CFTR) gene. Over the last 30 years, tremendous …

[HTML][HTML] Carrier capability of halloysite nanotubes for the intracellular delivery of antisense PNA targeting mRNA of neuroglobin gene

AP Falanga, M Massaro, N Borbone… - Journal of Colloid and …, 2024 - Elsevier
Peptide nucleic acid (PNA) is a DNA mimic that shows good stability against nucleases and
proteases, forming strongly recognized complementary strands of DNA and RNA. However …

Precise targeting of miRNA sites restores CFTR activity in CF bronchial epithelial cells

C De Santi, EF Fernández, R Gaul, S Vencken… - Molecular Therapy, 2020 - cell.com
MicroRNAs that are overexpressed in cystic fibrosis (CF) bronchial epithelial cells (BEC)
negatively regulate CFTR and nullify the beneficial effects of CFTR modulators. We …

Nanocarrier based on halloysite and fluorescent probe for intracellular delivery of peptide nucleic acids

M Massaro, E Licandro, S Cauteruccio… - Journal of colloid and …, 2022 - Elsevier
The development of systems able to deliver genetic material into a target site is a challenge
for modern medicine. Single-stranded peptide nucleic acids have attracted attention as …

CFTR dysfunction in cystic fibrosis and chronic obstructive pulmonary disease

E Fernandez Fernandez, C De Santi… - Expert review of …, 2018 - Taylor & Francis
Introduction: Obstructive lung diseases such as cystic fibrosis (CF) and chronic obstructive
pulmonary disease (COPD) are causes of high morbidity and mortality worldwide. CF is a …

Development of surface chemical strategies for synthesizing redox‐responsive diatomite nanoparticles as a green platform for on‐demand intracellular release of an …

M Terracciano, F Fontana, AP Falanga, S D'Errico… - Small, 2022 - Wiley Online Library
Redox‐responsive silica drug delivery systems are synthesized by aeco‐friendly diatomite
source to achieve on‐demand release of peptide nucleic acid (PNA) in tumor reducing …

Biopolymer-based nanoparticles for cystic fibrosis lung gene therapy studies

E Fernández Fernández, B Santos-Carballal… - Materials, 2018 - mdpi.com
Lung gene therapy for cystic fibrosis disease has not been successful due to several
challenges such as the absence of an appropriate vector. Therefore, optimal delivery of …

Use of adenine base editing and homology-independent targeted integration strategies to correct the cystic fibrosis causing variant, W1282X

K Mention, K Cavusoglu-Doran, AT Joynt… - Human molecular …, 2023 - academic.oup.com
Small molecule drugs known as modulators can treat~ 90% of people with cystic fibrosis
(CF), but do not work for premature termination codon variants such as W1282X (c. 3846G> …