Results 191 to 200 of about 323,927 (286)

Blood SOD1 Activity in ALS Patients Receiving Tofersen Treatment

open access: yesAnnals of Neurology, EarlyView.
Objective The antisense oligonucleotide tofersen is the first disease‐modifying drug for SOD1‐related amyotrophic lateral sclerosis (ALS) and was approved because of its ability to reduce SOD1 protein and neurofilament levels. The effect of tofersen on SOD1 activity is unclear but of clinical relevance because homozygous SOD1 mutations, linked to ...
Katharina Goehring   +18 more
wiley   +1 more source

Molecular Basis for Activation to Inhibition Switching in Kv7.2 Channel Modulators

open access: yesAngewandte Chemie, EarlyView.
The paper describes the serendipitous discovery of chemical manipulation allowing the activator‐to‐inhibitor switching in Kv7.2 channel modulators. The molecular determinants driving this switch have been rationalized by multidisciplinary investigation encompassing synthetic and analytical chemistry, in silico methods, cryo‐EM analysis ...
Tania Ciaglia   +20 more
wiley   +2 more sources

Biophysical Characterization and Proteomic Analysis of Small Extracellular Vesicles Derived From Different Neural Cell Lines

open access: yesAdvanced NanoBiomed Research, EarlyView.
This study systematically compares small extracellular vesicles (sEVs) derived from four neural cell lines, revealing how cellular origin shapes vesicle biophysical properties and proteomic cargo. Distinct, lineage‐specific signatures linked to neuronal, astrocytic, and microglial functions are identified, highlighting the importance of cell source ...
Muhammad Waqas Salim   +4 more
wiley   +1 more source

Glutathione‐Responsive Acyl‐Modifications for Targeted RNA Decaging and Prolonged Protein Synthesis

open access: yesAngewandte Chemie, EarlyView.
The self‐immolation of disulfide‐based mRNA modifications in response to endogenous glutathione (GSH) promotes a gradual release of translatable mRNA within the cell, leading to improved nuclease resistance, tunable release properties, and a significant increase (up to 600%) of target protein production over time. This strategy offers an exciting proof
Mary E. Flood   +6 more
wiley   +2 more sources

Unraveling A4GALT Mechanism and Its Modulation With Adamantyl‐Galactosylceramide Analogues: Advancing Fabry Disease Therapeutic Strategies

open access: yesAngewandte Chemie, EarlyView.
A 310‐helix‐mediated conformational switch promotes a front‐face SNi‐like catalysis by human A4GALT. Mechanism‐guided design identifies AdaGalCer as a selective modulator of globotriaosylceramide (Gb3) biosynthesis, opening a clear route toward new Fabry disease therapeutics.
Nicky de Koster   +13 more
wiley   +2 more sources

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