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Development of novel therapeutic agents by inhibition of oncogenic microRNAs
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 …
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
Minimally invasive technologies that can sample and detect cell-free nucleic acid
biomarkers from liquid biopsies have recently emerged as clinically useful for early …
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 …
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 …
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 …
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 …
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 …
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 …
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 …
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 …
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> …
(CF), but do not work for premature termination codon variants such as W1282X (c. 3846G> …