Results 91 to 100 of about 79,581 (296)

Molecular Mobility of N‐Acetylgalactosamine‐Modified Cyclodextrins on a Polyrotaxane for Highly Efficient Liver Targeting of Antibody Chimeras and Genome‐Editing Ribonucleoproteins

Advanced Science, EarlyView.
Monovalent N‐acetylgalactosamine (GalNAc)‐modified polyrotaxane enables efficient liver targeting by utilizing ligand mobility. The sliding and rotating cyclic components i.e., cyclodextrin in the polyrotaxane dynamically cluster GalNAc moieties, thereby mimicking trivalent interactions with asialoglycoprotein receptors.
Toru Taharabaru, Keiichi Motoyama, Yuting Wen, Zhongxing Zhang, Xuehao Tian, Jun Li, Taishi Higashi   +6 more
wiley   +1 more source

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

Membrane Fusion‐Mediated Cytosolic Delivery of Threose Nucleic Acids via Homotypic Nanoparticles Overcomes Drug Resistance in Triple‐Negative Breast Cancer

Advanced Science, EarlyView.
This study introduces a biomimetic “nanofusion” platform that integrates the biostability of threose nucleic acids (TNA) with homotypic cell‐membrane cloaking to combat drug‐resistant TNBC. By leveraging a non‐canonical membrane‐fusion pathway for direct cytosolic delivery, the platform bypasses endosomal sequestration. To achieve potent AKT2 silencing
Wei Zheng, Tristan Juin Han Chang, Xinchao Li, Zhongqi Zhou, Chung Tin, Kenward Vong, Pierre Karam, Pik Kwan Lo   +7 more
wiley   +1 more source

Rethinking antisense oligonucleotide therapeutics for amyotrophic lateral sclerosis

Annals of Clinical and Translational Neurology
Antisense oligonucleotides, which are used to silence target genes, are gaining attention as a novel drug discovery modality for proteinopathies. However, while clinical trials for neurodegenerative diseases like amyotrophic lateral sclerosis have been ...
Daisuke Ito, Kensuke Okada
doaj   +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

Antisense oligonucleotides targeted to human cdc45 [PDF]

, 2008
Antisense oligonucleotides that inhibit expression of human replication-initiation protein as well as methods of preventing or treating hyperproliferative conditions using said oligonucleotides are introduced.
Liang, Chun, Feng, Dao-rong, Yu, Zhiling
core  

Inhibition of KDEL Receptors Remodels the Tumor Microenvironment for T Cell Independent Tumor Regression

Advanced Science, EarlyView.
Inhibition of KDELR2 in a small fraction of tumor cells generates sustainable immunogenic cell death conditions within the tumor microenvironment. These conditions promote the regression of tumors in a T cell independent manner. During regression, macrophages prime T cells that subsequently provide systemic protection against recurrence. The potency of
Shakti P Pattanayak, Hong Wang, Belinda Willard, Timothy A. Chan, William C Merrick, Zheng‐Rong Lu, Boaz Tirosh   +6 more
wiley   +1 more source

Natural Variation of COLD and CATECHINS REGULATOR 1 Coordinately Fine‐Tunes Cold Tolerance and Tea Quality in Tea Plants

Advanced Science, EarlyView.
Multi‐trait genome‐wide association mapping identifies a central hub regulator, COLD AND CATECHINS REGULATOR 1 (CCR1), and its excellent natural allele variation, coordinately enhancing cold tolerance and promoting catechins biosyntheis. CsCCR1 interacts with CsCBF1/3 and is transcriptionally activated by CsLUX and CsKUA1 to promote catechins ...
Yanli Wang, Wei Tong, Qiong Wu, Didi Wu, Yanrui Zhang, Qijuan Gao, Fangdong Li, Zhaoliang Zhang, Wenjie Wang, Chuankui Song, Enhua Xia   +10 more
wiley   +1 more source

Non-antisense cellular responses to oligonucleotides [PDF]

, 2002
Oligonucleotides induce various cellular responses which are not due to the blockade of protein synthesis by an antisense mechanism. Oligonucleotides presenting double-stranded or G-quartet structures (ribo- or deoxyribonucleotides, phosphodiester or ...
Massoulié, Jean, Stefan Wietek, Bernard Payrastre, Anselmet, Alain, Ebrahim Mayat, Jean Massoulié, Mayat, Ebrahim, Payrastre, Bernard, Paul G Layer, Layer, Paul G, Wietek, Stefan, Alain Anselmet, Layer, Paul G.   +12 more
core   +1 more source

A Machine Vision‐Guided Microphysiological Platform With Automated Microfluidics Enables Longitudinal Biomarker Monitoring and Emulation of Translationally Relevant Exposure Scenarios

Advanced Science, EarlyView.
A pneumatically actuated multi‐tissue microphysiological system is integrated with AI‐based machine vision and automatic sampling and replenishment systems. The platform allows for the emulation of translationally relevant long‐term pharmacokinetic exposure scenarios for multiple weeks while enabling longitudinal monitoring of response biomarkers ...
Jibbe Keulen, Yi Zhong, Laura Dangel, Sonia Youhanna, Reza Zandi Shafagh, Jinhye Ryu, Domnica‐Gabriela Gurau, Mathias Haag, Thomas E. Mürdter, Yingxin Liang, Iro Beligiani, Nicole Ziegler, Sabine Willems, Nayere Taebnia, Wouter van der Wijngaart, Volker M. Lauschke   +15 more
wiley   +1 more source