Results 131 to 140 of about 186,376 (313)
Advanced Science, EarlyView.Molecular, genetic, virological, and biochemical analysis in combination with global proteome and phosphoproteome profiling and functional assays were applied to study the role of PR130 in the context of HSV‐1 replication. The observations reveal that host‐intrinsic mechanisms regulate HSV‐1 replication and highlight PR130 as a susceptibility factor of
Johannes Jungwirth +10 more
wiley +1 more source
Case control analysis of repeat expansion size in ataxia
, 2007 Spinocerebellar ataxias (SCAs) are a group of clinically and genetically heterogeneous neurological diseases. The expansion of unstable microsatellite repeats has been identified as the underlying pathogenic cause of 10 subtypes of autosomal dominant ...
Cross, W. +6 more
core +1 more source
Domain Specific Placebo Response in the Modified Friedreich's Ataxia Rating Scale
Annals of Clinical and Translational NeurologyThe placebo response in clinical trials in ataxias complicates outcome interpretation and potentially obscures genuine treatment effects. We analyzed placebo group data from past trials in Friedreich Ataxia and observed notable responses in appendicular ...
Christian Rummey +2 more
doaj +1 more source
Noninvasive Focal Gene Delivery into the Cerebellum of Non‐Human Primates using Focused Ultrasound
Advanced Science, EarlyView.Focal and non‐invasive viral vector delivery in non‐human primates remains a major challenge in translational neuroscience. Low‐intensity focused ultrasound was used to transiently open the blood–brain barrier and enable targeted gene delivery to the cerebellum.
Noelia Esteban‐García +11 more
wiley +1 more source
Nucleic Acid Therapeutics for “Undruggable” Cancer Targets: Mechanisms, Challenges, and Prospects
Advanced Science, EarlyView.Nucleic acid therapeutics bypass the structural limitations of conventional drugs by targeting mRNA rather than proteins. This review examines how antisense oligonucleotides, siRNAs, miRNAs, aptamers, and mRNA vaccines intervene against historically undruggable oncoproteins including Ras, MYC, and p53, highlighting mechanistic advances, delivery ...
Feng Xu +6 more
wiley +1 more source
Accelerating Biosensor Discovery: A Computationally‐Driven Pipeline for Microplastics Monitoring
Advanced Intelligent Discovery, EarlyView.A computationally guided pipeline unites molecular simulation, synthetic biology, electrochemical engineering, and machine learning to accelerate biosensor discovery. A Bacillus anthracis carbohydrate‐binding module is used to develop a high‐performance micro‐ and nanoplastics sensor with greatly reduced error and variability.
Gabriel X. Pereira +13 more
wiley +1 more source
Ataxia telangiectasia bemutatása egy eset kapcsán
, 2017 Az ataxia telangiectasia egy nagyon heterogén és ritka kórkép, melynek incidenciája 1:20000. Az Egyesült Államokban élő kaukázusi populáció 1,4-2 %-a hordozó.
Bunkóczi, Csilla
core
Frontiers in Human NeuroscienceThere is a critical need for accessible neuropsychological testing for basic research and translational studies worldwide. Traditional in-person neuropsychological studies are inherently difficult to conduct because testing requires the recruitment and ...
Sharon Binoy +6 more
doaj +1 more source
American Journal of Hematology, EarlyView.ABSTRACT Talquetamab is a GPRC5D × CD3 bispecific antibody approved for relapsed/refractory multiple myeloma (RRMM). This is the first real‐world study to comprehensively report muco‐cutaneous toxicities, infections, and efficacy of talquetamab in 151 patients across five countries with the longest follow‐up reported enabling better efficacy and ...
Murali Janakiram +94 more
wiley +1 more source
, 2012 Spinocerebellar Ataxia Type 2 (SCA2) is caused by expansion of a polyglutamine encoding triplet repeat in the human ATXN2 gene beyond (CAG)31. This is thought to mediate toxic gain-of-function by protein aggregation and to affect RNA processing ...
Damrath, Ewa +26 more
core +1 more source