Results 71 to 80 of about 127,002 (321)

Antisense Oligonucleotide-Mediated Removal of the Polyglutamine Repeat in Spinocerebellar Ataxia Type 3 Mice

Molecular Therapy: Nucleic Acids, 2017
Spinocerebellar ataxia type 3 (SCA3) is a currently incurable neurodegenerative disorder caused by a CAG triplet expansion in exon 10 of the ATXN3 gene.
Lodewijk J.A. Toonen, Frank Rigo, Haico van Attikum, Willeke M.C. van Roon-Mom   +3 more
doaj   +1 more source

Author Correction: Antisense oligonucleotide development for the selective modulation of CYP3A5 in renal disease [PDF]

, 2021
Kevin A. Lidberg, Andrew J. Annalora, Marija Jozic, Daniel J. Elson, Lu Wang, Theo K. Bammler, Susanne Ramm, Maria Beatriz Monteiro, Jonathan Himmelfarb, Craig B. Marcus, Patrick L. Iversen, Edward J. Kelly   +11 more
openalex   +1 more source

From RNA to DNA: How Cargo Identity Reprograms Lipid Nanoparticle Architecture and Function

Advanced Healthcare Materials, EarlyView.
The evolution of lipid nanoparticles (LNPs) spans from RNA‐LNPs, used in mRNA vaccines, to DNA‐LNPs, ideal for gene therapies. Emerging bionano architectures, decorated with DNA and plasma proteins, pave the way for advanced DNA‐based therapies that are more stable, targeted, and customizable.
Erica Quagliarini, Daniela Pozzi, Giulio Caracciolo   +2 more
wiley   +1 more source

Cathepsin B-activatable cyclic antisense oligonucleotides for cell-specific target gene knockdown in vitro and in vivo

Molecular Therapy: Nucleic Acids, 2023
Trigger-activatable antisense oligonucleotides have been widely applied to regulate gene function. Among them, caged cyclic antisense oligonucleotides (cASOs) maintain a specific topology that temporarily inhibits their interaction with target genes.
Zhongyu Wang, Xinli Fan, Guanqun Mu, Xiaoran Zhao, Qian Wang, Jing Wang, Xinjing Tang   +6 more
doaj   +1 more source

Antisense PNA accumulates in Escherichia coli and mediates a long post-antibiotic effect [PDF]

, 2007
Antisense agents that target growth-essential genes display surprisingly potent bactericidal properties. In particular, peptide nucleic acid (PNA) and phosphorodiamidate morpholino oligomers linked to cationic carrier peptides are effective in time kill ...
Behmanesh, Mehrdad, Dryselius, Rikard, Faridani, Omid R, Ganyu, Anita, Goh, Shan, Good, Liam, Klok, Erik Jan, Nikravesh, Abbas, Sadeghizadeh, Majid, Zain, Rula   +9 more
core   +2 more sources

Potent Liver‐Tropic mRNA Lipid Nanoparticles: ApoE‐Mediated Delivery Through a Low‐Density Lipoprotein Receptor Independent Uptake Mechanism

Advanced Materials, EarlyView.
Helper and ionizable lipids play a crucial role in determining ApoE binding and subsequent liver tropism and LDLR‐mediated uptake. Ionizable lipids primarily govern the LDLR‐independent uptake pathway. This complementary interplay between lipid components ultimately governs LNP delivery performance and therapeutic efficacy in the liver.
Ashish Sarode, Christian Ortiz, Tadeh Derstepanian, Natalia Vargas‐Montoya, Priyal Patel, Nikita Khadse, Saikat Manna, Ryan Landis, Joseph Skaleski, Lianne Boeglin, Hongfeng Deng, Anusha Dias, Hong Wang, Debora Barreiros Petropolis, Barak Yahalom, Shrirang Karve, Frank DeRosa   +16 more
wiley   +1 more source

Position of Premature Termination Codons Determines Susceptibility of hERG Mutations to Nonsense-Mediated mRNA Decay in Long QT Syndrome

, 2014
The degradation of human ether-a-go-go-related gene (hERG, KCNH2) transcripts containing premature termination codon (PTC)mutations by nonsense-mediatedmRNA decay (NMD) is an importantmechanismof long QT syndrome type 2 (LQT2).
Gong, Qiuming, Stump, Matthew R., Zhou, Zhengfeng   +2 more
core   +1 more source

CCDC41 Drives Oocyte Meiotic Progression by Promoting Rab11a/Rab7‐Positive Vesicle Fusion with Target Membranes

Advanced Science, EarlyView.
CCDC41 is essential for meiotic maturation in mouse oocytes through regulating Rab7‐positive endosomes fusion with lysosomes and Rab11a‐positive vesicle fusion with the plasma membrane. Abstract Coiled‐coil domain‐containing protein 41 (CCDC41), a core component of centriolar distal appendages involved in centriole assembly and ciliary vesicle docking,
Ying Tian, Jiatong Li, Jingyi Kang, Xiangning Xu, Bicheng Wang, Shuo Lou, Jingyu Li, Yuying Yang, Yanbing Zhang, Yangzi Zheng, Jing Weng, Yuanjing Liang, Wei Ma   +12 more
wiley   +1 more source

Formation of an RNA Quadruplex-Duplex Hybrid in Living Cells between mRNA of the Epidermal Growth Factor Receptor (EGFR) and a G-Rich Antisense Oligoribonucleotide

Cells, 2020
Antisense DNA oligonucleotides, short interfering RNAs (siRNAs), and CRISPR/Cas9 genetic tools are the most useful therapeutic nucleic acids regulating gene expression based on the antisense specificity towards messenger RNA.
Dorota Gudanis, Damian Kaniowski, Katarzyna Kulik, Daniel Baranowski, Zofia Gdaniec, Barbara Nawrot   +5 more
doaj   +1 more source

Antisense oligonucleotide induction of progerin in human myogenic cells. [PDF]

PLoS ONE, 2014
We sought to use splice-switching antisense oligonucleotides to produce a model of accelerated ageing by enhancing expression of progerin, translated from a mis-spliced lamin A gene (LMNA) transcript in human myogenic cells. The progerin transcript (LMNA
Yue-Bei Luo, Chalermchai Mitrpant, Abbie M Adams, Russell D Johnsen, Sue Fletcher, Frank L Mastaglia, Steve D Wilton   +6 more
doaj   +1 more source