Results 41 to 50 of about 959,158 (292)

Cognitive and Neuroimaging Divergence Between Juvenile and Adult FUS Amyotrophic Lateral Sclerosis

Annals of Clinical and Translational Neurology, EarlyView.
ABSTRACT Objective Amyotrophic lateral sclerosis (ALS) is a neurodegenerative disorder characterized by progressive motor neuron degeneration. Fused in sarcoma (FUS)‐associated juvenile ALS (jALS) represents a distinct and aggressive subgroup with rapid deterioration and poor prognosis.
Alexandra V. Jürs, Marcel Naumann, Annaliis Lehto, Hanna Schön, Jens Kurth, Johannes Prudlo, Andreas Hermann, Elisabeth Kasper   +7 more
wiley   +1 more source

Gene isoform specificity through enhancer-associated antisense transcription.

PLoS ONE, 2012
Enhancers and antisense RNAs play key roles in transcriptional regulation through differing mechanisms. Recent studies have demonstrated that enhancers are often associated with non-coding RNAs (ncRNAs), yet the functional role of these enhancer:ncRNA ...
Courtney S Onodera, Jason G Underwood, Sol Katzman, Frank Jacobs, David Greenberg, Sofie R Salama, David Haussler   +6 more
doaj   +1 more source

Integrated PANoptosis Profiling Identifies Immunosuppressive Subtypes and a Prognostic Signature With Functional Validation of MLKL in Glioblastoma

Annals of Clinical and Translational Neurology, EarlyView.
ABSTRACT Objective The prognosis of glioblastoma (GBM) remains highly unfavorable, largely due to high tumor heterogeneity and an immunosuppressive microenvironment. However, the functional role of PANoptosis in this context is poorly understood. Methods Patients were stratified via K‐means clustering. A risk score model was constructed using prognosis‐
Langfei Tian, Minghui Zhao, Yuanbo Hu, Kaiyue Wang, Haiguang Liu, Zetong Bai, Kebin Zheng   +6 more
wiley   +1 more source

Silencing disease genes in the laboratory and the clinic

, 2011
Synthetic nucleic acids are commonly used laboratory tools for modulating gene expression and have the potential to be widely used in the clinic. Progress towards nucleic acid drugs, however, has been slow and many challenges remain to be overcome before
Jonathan K Watts, David R Corey, Corey, David R., Watts, Jonathan K.   +3 more
core   +1 more source

Augmenting and Assaying Nav1.1 Protein Quantity for Dravet Syndrome Therapy

Annals of Clinical and Translational Neurology, EarlyView.
ABSTRACT Dravet Syndrome (DS) is a developmental and epileptic encephalopathy predominantly caused by heterozygous loss‐of‐function variants in SCN1A, which encodes Nav1.1. Conserved upstream open reading frames (uORFs) in SCN1A were validated to regulate translation in reporter assays, demonstrating the therapeutic viability of increasing Nav1.1 from ...
Aiswarya Saravanan, Jordan Safran, Anna Loughman, Grace Lin, Shivanshi Vaid, Dana Allababidi, Peter M. Tessier, Louis T. Dang   +7 more
wiley   +1 more source

Hitting bacteria at the heart of the central dogma: sequence-specific inhibition

Microbial Cell Factories, 2007
An important objective in developing new drugs is the achievement of high specificity to maximize curing effect and minimize side-effects, and high specificity is an integral part of the antisense approach.
Mortensen Kim, Sperling-Petersen Hans, Rasmussen Louise   +2 more
doaj   +1 more source

RNA Interference by Single- and Double-stranded siRNA With a DNA Extension Containing a 3′ Nuclease-resistant Mini-hairpin Structure [PDF]

, 2013
Selective gene silencing by RNA interference (RNAi) involves double-stranded small interfering RNA (ds siRNA) composed of single-stranded (ss) guide and passenger RNAs.
Jo Milner, Simon J Allison, Milner, J., Allison, Simon J., Milner, Jo   +4 more
core   +1 more source

Fusogenic RNA Nanomodules for Fusion‐Mediated and Multiplexed siRNA Delivery

Advanced Functional Materials, EarlyView.
A fusogenic lipid‐layered RNA nanomodules (L‐CRAMs) enable high‐capacity and long‐lasting siRNA delivery through membrane fusion. These nanomodules carry exceptionally large siRNA payloads, avoid conventional endosomal uptake, and release multiple functional siRNAs through Dicer‐mediated processing.
Sunghyun Moon, Iksoo Jang, Taehyeon Kim, Nayoung Yoon, Kyuha Yum, Jong Bum Lee   +5 more
wiley   +1 more source

Viral escape from antisense RNA [PDF]

Molecular Microbiology, 1998
RNA coliphage SP was propagated for several generations on a host expressing an inhibitory antisense RNA complementary to bases 31–270 of the positive‐stranded genome. Phages evolved that escaped inhibition. Typically, these escape mutants contained 3–4 base substitutions, but different sequences were observed among different isolates.
J J, Bull, A, Jacobson, M R, Badgett, I J, Molineux   +3 more
openaire   +2 more sources

Nano Borophene as a Programmable 2D Scaffold for Organized DNA Assembly and Long‐Range Energy Transfer

Advanced Functional Materials, EarlyView.
Borophene is introduced as a programmable 2D bio‐interface that organizes DNA through dual boron–sulfur and nucleobase interactions. This molecular architecture enables efficient long‐range nanosurface energy transfer, establishing new design principles for functional nano–bio interfaces and amplification‐free optical biosensing.
Teresa Aditya, Pranay Saha, John M. Sansalone, Shraddha Krishnakumar, Matthew Molinaro, André J. van der Vlies, Nada Maher, Adrienne Griffiths, Zakhro Zakirova, Ketan Dighe, Narayana R. Aluru, Dipanjan Pan   +11 more
wiley   +1 more source