Results 81 to 90 of about 143,834 (286)

The Dynamics of Compound, Transcript, and Protein Effects After Treatment With 2OMePS Antisense Oligonucleotides in mdx Mice

Molecular Therapy: Nucleic Acids, 2014
Antisense-mediated exon skipping is currently in clinical development for Duchenne muscular dystrophy (DMD) to amend the consequences of the underlying genetic defect and restore dystrophin expression. Due to turnover of compound, transcript, and protein,
Ingrid E C Verhaart, Laura van Vliet-van den Dool, Jessica A Sipkens, Sjef J de Kimpe, Ingrid G M Kolfschoten, Judith C T van Deutekom, Lia Liefaard, Jim E Ridings, Steve R Hood, Annemieke Aartsma-Rus   +9 more
doaj   +1 more source

circZNF148 Drives Glucose Metabolism Reprogramming to Enhance Metastasis and Immune Evasion via HK1 Stabilization in Triple‐Negative Breast Cancer

Advanced Science, EarlyView.
CircZNF148 stabilizes HK1 through deubiquitinase recruitment, thereby enhancing glycolysis and lactate production. Elevated lactate promotes PD‑L1 lactylation and membrane accumulation while suppressing CD8+ T‐cell cytotoxicity, collectively facilitating immune evasion and malignant progression in TNBC.
Yuhan Jin, Wenjing Zhao, Lei Wang, Jianing Wang, Keying Zhao, Dan Luo, Yifei Wang, Yuxia Yang, Jingze Yang, Yaming Li, Tong Chen, Dianwen Han, Zekun Wang, Xiaoyan Li, Bing Chen, Lijuan Wang, Yiran Liang, Qifeng Yang   +17 more
wiley   +1 more source

Antisense oligonucleotides and their applications in rare neurological diseases

Frontiers in Neuroscience
Rare diseases affect almost 500 million people globally, predominantly impacting children and often leading to significantly impaired quality of life and high treatment costs. While significant contributions have been made to develop effective treatments
Simon McDowall, Simon McDowall, May Aung-Htut, May Aung-Htut, Steve Wilton, Steve Wilton, Dunhui Li, Dunhui Li   +7 more
doaj   +1 more source

Sintesi e Purificazione di Oligonucleotidi e Nucleosidi 2’- funzionalizzati [PDF]

, 2009
Oligonucleotides (ON) are commonly used as research reagents to modulate gene expression in cell culture and in animal models. Various chemistries of ON have been synthesized, incorporating modifications to the phosphodiester backbone, sugar or ...
Mari, Lara
core  

Biodistribution of Ga-68-labeled LNA-DNA mixmer antisense oligonucleotides for rat Chromogranin-A

, 2008
In vivo monitoring of gene expression may be accomplished using a most advanced imaging technology such as positron emission tomography (PET). However, a range of methodological and biological hurdles needs exploration.
Bergström, Mats,, Eriksson, Barbro,, Lendvai, Gabor, Estrada, Sergio, Velikyan, Irina, Långström, Bengt,   +5 more
core   +1 more source

A Small‐Molecule DEPTAC Rescues Cognitive Deficits by Targeted Dephosphorylation of Pathological Tau

Advanced Science, EarlyView.
TP2 is a fully synthetic small‐molecule dephosphorylation‐targeting chimera that recruits endogenous PP2A‐Bα to Tau, enabling targeted removal of pathological phosphate modifications. In two tauopathy mouse models, systemic TP2 reduces Tau pathology, preserves neuronal and synaptic integrity, improves neuroimmune homeostasis, and rescues cognition ...
Fei Sun, Yang Liu, Qiuzhi Zhou, Qingyuan Hu, Weixia Wang, Yue Xiao, Jingfen Su, Guanting Liu, Miaomiao Sun, Lixia Chen, Hua Li, Ying Yang, Jian‐Zhi Wang   +12 more
wiley   +1 more source

Antisense oligonucleotides in neurological disorders

Therapeutic Advances in Neurological Disorders, 2018
The introduction of genetics revolutionized the field of neurodegenerative and neuromuscular diseases and has provided considerable insight into the underlying pathomechanisms. Nevertheless, effective treatment options have been limited.
Claudia D. Wurster, Albert C. Ludolph
doaj   +1 more source

A Cascaded DNA Nanocircuit for Multi‐Signal‐Responsive Precision siRNA Delivery in Cancer Therapy

Advanced Science, EarlyView.
A cascaded dual‐AND logic DNA nanocircuit is engineered to respond to three tumor‐specific signals in a sequential manner: extracellular acidic pH, membrane‐overexpressed nucleolin (NCL), and intracellular glutathione (GSH). This programmable system selectively releases siPARP1 in glioblastoma (GBM), effectively silencing PARP1 and reversing TMZ ...
Yan Zhao, Yufei Lan, Min‐Goo Lee, Jinjun He, Yueran Pan, Eunji Kim, Xin Fu, Sungwook Jung, Wanxia Lu, Jinyue Fan, Haoxiang Chen, Yuanbo Zhao, Yuling Xu, Jong Seung Kim, Chao Zhang   +14 more
wiley   +1 more source

In Vivo Direct Reprogramming: Current Progress and Future Prospects from Mechanisms to Therapeutic Application

Advanced Science, EarlyView.
This review examines the potential of in vivo direct reprogramming in regenerative medicine for functional tissue restoration, highlighting the role of tissue‐resident cues in generating functionally mature reprogrammed cells from lineage‐related cells. It contains a discussion on mechanisms, reprogramming factors, delivery approaches, and applications
Rishabh Deo Singh, Mauro Calvoli, Kyeong Kyu Kim   +2 more
wiley   +1 more source

Restoration of m6A‐Modified circPSMB1 Suppresses Chordoma Progression and Enables Local Nanotherapeutic Intervention

Advanced Science, EarlyView.
An m6A‐modified circRNA, circPSMB1, is identified as a tumor suppressor in chordoma. METTL3‐dependent m6A promotes circPSMB1 interaction with HNRNPA2B1, limiting c‐MYC expression and malignant progression. An injectable HMnO2‐based nanohydrogel enables local circPSMB1 delivery and tumor microenvironment‐responsive therapy, offering a promising strategy
Yingchuang Tang, Liang Qiu, Xingbang Ruan, Yihan Shi, Ye Wang, Hanwen Li, Kai Zhang, Kangwu Chen   +7 more
wiley   +1 more source