Results 61 to 70 of about 143,834 (286)

Repair of Aberrant Splicing in Growth Hormone Receptor by Antisense Oligonucleotides Targeting the Splice Sites of a Pseudoexon [PDF]

, 2010
Context: The GH receptor (GHR) pseudoexon 6 Psi defect is a frequent cause of GH insensitivity (GHI) resulting from a non-functioning GH receptor (GHR). It results in a broad range of phenotypes and may also be present in patients diagnosed as idiopathic
Srirangalingam, U, David, A, Metherell, LA, Khoo, B, Clark, AJL   +4 more
core  

Antisense-Induced Fas mRNA Degradation Produces Site-Specific Stable 3'-mRNA Fragment by Exonuclease Cleavage at the Complementary Sequence

, 2004
Antisense-mediated degradation of target mRNA is achieved by the enzymatic action of nuclease RNase H. The enzyme recognizes hybrid RNA-DNA duplexes and hydrolyzes the RNA strand.
Dahlgren, Cecilia, Thonberg, H,, Cecilia Dahlgren, Dahlgren, C, Wahlestedt, C, Claes Wahlestedt, Wahlestedt, Claes, Håkan Thonberg, Thonberg, Håkan   +8 more
core   +1 more source

Smart Nanotechnologies for Multimodal Neuromodulation and Brain Interfacing

Advanced Science, EarlyView.
Recent advances in smart nanotechnologies are expanding the toolbox for brain interfacing, from wireless neuromodulation and high‐resolution sensing to targeted delivery within the central nervous system. By combining responsive nanomaterials with bioinspired design, these platforms enable multimodal interactions with neurons and glia, while also ...
Tommaso Curiale, Marie Celine Lefevre, Alessio Carmignani, Maria Cristina Ceccarelli, Matteo Battaglini, Attilio Marino, Gianni Ciofani   +6 more
wiley   +1 more source

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

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

Investigation of the role of LRP in multidrug resistance [PDF]

, 1998
There have been many reports linking the overexpression of the lung resistance-related protein (LRP) with cross-resistance to chemotherapeutic drugs.
Byrne, Daragh
core   +2 more sources

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

Unraveling Bone‐Skin Crosstalk Enables miRNA Nanoformulation for Cutaneous Neurovascular Reconstruction in Diabetic Mice

Advanced Science, EarlyView.
This study unravels that bone fracture accelerates diabetic wound healing by releasing exosomal microRNA (miR‐130b‐3p). To mimic this bone‐skin crosstalk, we engineered a self‐assembling agomir‐130b‐3p nanocomplex. Delivered via a photocrosslinkable hydrogel, this bio‐inspired therapy provides sustained localized agomir release.
Tao Shen, Hongdou Shen, Weiyue Wu, Zhuoying Jiang, Bin Liu, Yansi Xian, Rui Peng, An Yan, Yu Ben, Yong Shi, Xiang Chen, Baosheng Guo, Qing Jiang, Weijun Wang, Pingqiang Cai   +14 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

Long-term administration of antisense oligonucleotides into the paraspinal muscles of mdx mice reduces kyphosis

, 2008
The mdx mouse model of muscular dystrophy has a premature stop codon preventing production of dystrophin. This results in a progressive phenotype causing centronucleation of skeletal muscle fibers, muscle weakness and fibrosis and kyphosis.
Laws, Nicola, Fletcher, Susan, Cornford-Nairn, Renee A., Wilton, Stephen D., Hoey, Andrew J., Irwin, Nicole, Johnsen, Russell   +6 more
core   +1 more source