Results 71 to 80 of about 94,605 (281)

Nap1L4a Cooperates with Scl/Klf1 to Recruit H2A.Z in Mediating Interactions Among Cis‐Regulatory Elements and Transcription Required for Primitive Erythropoiesis in Zebrafish

Advanced Science, EarlyView.
Nap1l4a is required in erythropoiesis and hypoxia responses via physical interaction with Klf1 and Scl to recruit the histone variant H2A.Z. This facilitates its associated cis‐regulatory element (CRE) remodeling and the consequent chromatin assembly, and activates the transcription of erythroid lineage‐specific genes.
JiaHao Shi, FuMing Lai, Zheng Shen, XiaoYan Zhang, HanFei Wang, WenYe Liu, YunLong Wang, KuanYu Li, GuoLiang Li, YaPing Fang, Jing‐Xia Liu   +10 more
wiley   +1 more source

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

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

Crossing the Blood–Brain Barrier with Molecularly Imprinted Polymeric Nanocarriers: An Emerging Frontier in Brain Disease Therapy

Advanced Science, EarlyView.
Molecularly imprinted polymeric nanocarriers (nanoMIPs) offer robust, antibody‐mimetic platforms to overcome the blood‐brain barrier. The article surveys nanoMIP design and ligand‐directed surface engineering that harness receptor‐mediated transcytosis, and highlights therapeutic and diagnostic applications in neurodegeneration, brain tumors and ...
Ranjit De, Shuliang Shi, Kyong‐Tai Kim
wiley   +1 more source

Transforming RNA Degradation into Accurate RNA Detection: RNase I–Assisted Rolling Circle Amplification for Clinical Samples

Angewandte Chemie, EarlyView.
Here, we transform this otherwise destructive enzymatic activity into a powerful diagnostic advantage through an RNase I–assisted rolling circle amplification (RI‐RCA) strategy. By integrating controlled RNase I–mediated RNA digestion with circular DNA templates, this approach enables direct and highly sensitive detection of target RNA sequences. Using
Amal Mathai, Cheng‐Yu Lee, Zijie Zhang, Jimmy Gu, John D. Brennan, Yingfu Li   +5 more
wiley   +2 more sources

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

Advanced 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, Xiao Zhang, Jinze Li, Ning Xu, Sheng Hu, Qiyu Lu, Yuxuan Li, Taifu Xiao, Xu Li, Xue Wang, Kequan Xu, Daoming Liang   +11 more
wiley   +1 more source

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

Advanced 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, Jiale Sun, Yuqing Mao, Yuhao Tang, Fuan Wang, Zhihong Liu, Jing Wang   +6 more
wiley   +1 more source

Tumor Extracellular Vesicles lncOSLMT Drives Lung Inflammatory Premetastatic Niche Formation in Osteosarcoma via m6A‐Dependent hnRNPA2B1/COX‐2 Axis

Advanced Science, EarlyView.
Tumor‐derived extracellular vesicles program inflammatory lung premetastatic niches through selective delivery of long noncoding RNAs. This work reveals EVs‐associated lncOSLMT as a key driver of lung fibroblast activation via m6A‐dependent PTGS2 stabilization.
Hongbo Li, Zehao Guo, Yutong Zou, Jiongfeng Zhang, Jixiang Shi, Xihong Fu, Jian Tu, Hao Yao, Xuanxuan Lu, Lili Wen, Xianbiao Xie   +10 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

A Sirtuin‐1‐Targeted Gene‐Activating Tetrahedral DNA Attenuates Bladder Fibrosis by Restoring Mitophagy in Fibroblasts via the SIRT1‐FOXO3‐BNIP3 Axis

Advanced Science, EarlyView.
The SIRT1‐targeted saRNA‐delivering tetrahedral DNA (TSA) treatment effectively upregulates SIRT1 expression, which subsequently promotes FOXO3A deacetylation. This deacetylation event relieves FOXO3A's transcriptional repression on the BNIP3 gene, thereby initiating PINK1‐PARKIN‐dependent mitophagy.
Wei Wang, Ran Yan, Lede Lin, Lei Xiang, Xiaozhi Xia, Liao Peng, Xiaoshuai Gao, Jiawei Chen, Yang Xiong, Liang Zhou, Yunfeng Lin   +10 more
wiley   +1 more source