Results 81 to 90 of about 171,175 (241)

ISS-N1 makes the first FDA-approved drug for spinal muscular atrophy

Translational Neuroscience, 2017
Spinal muscular atrophy (SMA) is one of the leading genetic diseases of children and infants. SMA is caused by deletions or mutations of Survival Motor Neuron 1 (SMN1) gene.
Ottesen Eric W.
doaj   +1 more source

Metabolite Profiling of the Antisense Oligonucleotide Eluforsen Using Liquid Chromatography-Mass Spectrometry

Molecular Therapy: Nucleic Acids, 2019
Eluforsen (previously known as QR-010) is a 33-mer 2′-O-methyl modified phosphorothioate antisense oligonucleotide targeting the F508del mutation in the gene encoding CFTR protein of cystic fibrosis patients.
Jaeah Kim, Babak Basiri, Chopie Hassan, Carine Punt, Erik van der Hage, Cathaline den Besten, Michael G. Bartlett   +6 more
doaj   +1 more source

Inhibition of EGF Uptake by Nephrotoxic Antisense Drugs In Vitro and Implications for Preclinical Safety Profiling

Molecular Therapy: Nucleic Acids, 2017
Antisense oligonucleotide (AON) therapeutics offer new avenues to pursue clinically relevant targets inaccessible with other technologies. Advances in improving AON affinity and stability by incorporation of high affinity nucleotides, such as locked ...
Annie Moisan, Marcel Gubler, Jitao David Zhang, Yann Tessier, Kamille Dumong Erichsen, Sabine Sewing, Régine Gérard, Blandine Avignon, Sylwia Huber, Fethallah Benmansour, Xing Chen, Roberto Villaseñor, Annamaria Braendli-Baiocco, Matthias Festag, Andreas Maunz, Thomas Singer, Franz Schuler, Adrian B. Roth   +17 more
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

Schematic of the tandem antisense oligonucleotide approach.

, 2015
WT and Mut mRNA can interact with the gapmer or inhibitor, resulting in the formation of the most favorable thermodynamically duplexes (WT RNA/inhibitor and Mut RNA/gapmer).
Jolanta Lisowiec-Wachnicka (823327), Elzbieta Kierzek (27228), Ewa Biala (823326), Ryszard Kierzek (27233), Dorota Magner (722713)   +4 more
core   +1 more source

Glucose and Glutamine Deprivation Promotes Breast Cancer Lung Metastasis via BRD4‐Dependent Enhancer Activation

Advanced Science, EarlyView.
To model a nutrient‐restricted tumor microenvironment, breast cancer cells were adapted to low glucose and glutamine levels. In the adapted cells, BRD4‐driven enhancer activation increases pro‐invasive EDN1 expression, facilitated by ATF3/c‐JUN and reinforced by enhancer RNA.
Poshan Yugal Bhattarai, Arathy Vasukutty, Yujin Na, Sung‐Chul Lim, Hong Seok Choi   +4 more
wiley   +1 more source

Antisense Oligonucleotide Gene Therapy for Neuromuscular Disorders

University of Ottawa Journal of Medicine, 2018
Antisense oligonucleotides (ASOs) are synthetic, single-stranded DNA molecules that can bind to specific mRNA sequences and alter protein expression.
Ryan Gotesman
doaj   +1 more source

Cancer‐Associated Fibroblasts Promote Glucose Metabolic Reprogramming and Progression of Triple‐Negative Breast Cancer via Exosomal circFAD104‐Mediated Intercellular Communication

Advanced Science, EarlyView.
CAF‐derived exosomes deliver circFAD104 into TNBC cells, where it acts as a molecular scaffold that bridges the E3 ligase MARCHF8 and PGM1, promoting MARCHF8‐mediated K48‐linked ubiquitination and proteasomal degradation of PGM1. Loss of PGM1 redirects glucose‐phosphate flux from glycogen synthesis toward glycolysis, thereby driving stemness, EMT, and ...
Lei Wang, Xiaoyan Li, Yiran Liang, Yifei Wang, Yuhan Jin, Jianing Wang, Dan Luo, Yuxia Yang, Jingze Yang, Yaming Li, Tong Chen, Dianwen Han, Zekun Wang, Wenjing Zhao, Bing Chen, Lijuan Wang, Qifeng Yang   +16 more
wiley   +1 more source