Results 121 to 130 of about 1,703,096 (348)

Simultaneous Down-Regulation of Intracellular hTERT and GPX4 mRNA Using MnO2-Nanosheet Probes to Induce Cancer Cell Death

open access: yesSensors
Cancer remains a leading global cause of death, with conventional treatments often limited by toxicity and recurrence. Recent advances in gene therapy and nanodrug delivery offer new avenues for precision oncology. Human telomerase reverse transcriptase (
Yixin Miao   +3 more
doaj   +1 more source

CRISPLD2 Attenuates Intervertebral Disc Degeneration by Suppressing Oxidative Stress‐Induced Ferroptosis through the miR‐548I‐IL17A Axis

open access: yesAdvanced Science, EarlyView.
This study identifies CRISPLD2 as a key protector against IVDD. By regulating ferroptosis through the CRISPLD2–miR‐548I–IL17A axis, CRISPLD2 maintains NPCs homeostasis and reduces oxidative stress. Restoring CRISPLD2 expression effectively alleviates disc degeneration and highlights a promising therapeutic strategy for discogenic low back pain ...
Yangyang Shi   +11 more
wiley   +1 more source

Dual-targeting CRISPR-CasRx reduces C9orf72 ALS/FTD sense and antisense repeat RNAs in vitro and in vivo

open access: yesNature Communications
The most common genetic cause of frontotemporal dementia (FTD) and amyotrophic lateral sclerosis (ALS) is an intronic G4C2 repeat expansion in C9orf72.
Liam Kempthorne   +29 more
doaj   +1 more source

Antisense and Functional Nucleic Acids in Rational Drug Development

open access: yesAntibiotics
This review is focused on antisense and functional nucleic acid used for completely rational drug design and drug target assessment, aiming to reduce the time and money spent and increase the successful rate of drug development. Nucleic acids have unique
Robert Penchovsky   +4 more
doaj   +1 more source

The Use of Tricyclo-DNA Oligomers for the Treatment of Genetic Disorders

open access: yesBiomedicines, 2017
Antisense Oligonucleotides (ASOs) represent very attractive therapeutic compounds for the treatment of numerous diseases. The antisense field has remarkably progressed over the last few years with the approval of the first antisense drugs and with ...
Philippine Aupy   +3 more
doaj   +1 more source

OCTN2 Activates a Non‐Canonical Carnitine Metabolic Pathway to Promote MASH‐HCC Progression and Immunotherapy Resistance

open access: yesAdvanced Science, EarlyView.
In non‐MASH‐HCC, L‐carnitine promotes tumor progression primarily through its classical role in enhancing fatty acid oxidation (FAO). However, in MASH‐HCC, where FAO is markedly suppressed, L‐carnitine shifts from this canonical function to serve instead as an intracellular acetyl group buffer.
Chuqi Xia   +11 more
wiley   +1 more source

The Circadian Clock Gene Period1 Connects the Molecular Clock to Neural Activity in the Suprachiasmatic Nucleus. [PDF]

open access: yes, 2015
The neural activity patterns of suprachiasmatic nucleus (SCN) neurons are dynamically regulated throughout the circadian cycle with highest levels of spontaneous action potentials during the day.
Block, Gene D   +2 more
core   +1 more source

A Modular DNAzyme for Precise Visualization and Intervention of Alternative Splicing Isoforms in Live Cells

open access: yesAdvanced Science, EarlyView.
A Stringent dUPlex‐activated Error Robust (SUPER) DNAzyme system enables real‐time imaging of alternative mRNA splicing (e.g., Bcl‐xL/Bcl‐xS) in living cells via target‐triggered split‐DNAzyme reassembly and dual‐color fluorescence. It also achieves mRNA‐selective knockdown through DNAzyme‐based gene regulation, serving as a versatile tool for splicing
Mengru Lin   +6 more
wiley   +1 more source

Rescue of splicing-mediated intron loss maximizes expression in lentiviral vectors containing the human ubiquitin C promoter. [PDF]

open access: yes, 2014
Lentiviral vectors almost universally use heterologous internal promoters to express transgenes. One of the most commonly used promoter fragments is a 1.2-kb sequence from the human ubiquitin C (UBC) gene, encompassing the promoter, some enhancers, first
Cooper, Aaron R   +3 more
core   +1 more source

Synergistic Mn‐MOF Activation of Pistol Ribozymes for Cancer Immunotherapy

open access: yesAdvanced Science, EarlyView.
A Mn2+‐based metal–organic framework delivers a catalytic Pistol ribozyme for targeted PD‐L1 mRNA cleavage in tumor cells, with the released Mn2+ not only inducing immune activation in immune cells but also increasing the Pistol ribozyme catalytic activity.
Ming Zhao   +10 more
wiley   +1 more source

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