Results 131 to 140 of about 72,467 (253)

WONOEP appraisal: Biomarkers and treatment strategies beyond the synapse

Epilepsia, EarlyView.
Abstract Epilepsy is a heterogeneous neurological disorder affecting more than 70 million people worldwide, posing significant challenges for clinicians due to its complex etiology, diverse manifestations, variable treatment responses, and the inability to predict seizures or disease onset reliably.
Mirte Scheper, Zining Liu, Aristea S. Galanopoulou, Gernot Hlauschek, Benjamin Sinclair, Wai Lam Leung, Stefanie Dedeurwaerdere, Elisabeth Doran, Solomon L. Moshé, David C. Henshall, Ozlem Akman, Pablo M. Casillas‐Espinosa   +11 more
wiley   +1 more source

A xenotransplantation model for reactivation of paternal UBE3A using human-specific antisense oligonucleotides. [PDF]

Sci Rep
Smeenk H, Lendemeijer B, Buurma MG, Forgione MA, Slump DE, Monshouwer RA, Wallaard I, Mientjes EJ, Hoogendijk WJG, Elgersma Y, de Vrij FMS, Kushner SA.   +11 more
europepmc   +1 more source

Sodium Channel Blocker Tolerance as a Metric for Disease Modification in Dravet Syndrome

Annals of the Child Neurology Society, EarlyView.
Koichiro Yuji, Wakako Yuji
wiley   +1 more source

Genetic epilepsies with myoclonic seizures: Mechanisms and syndromes

Epilepsia Open, EarlyView.
Abstract Genetic epilepsy with myoclonic seizures encompasses a heterogeneous spectrum of conditions, ranging from benign and self‐limiting forms to severe, progressive disorders. While their causes are diverse, a significant proportion stems from genetic abnormalities.
Antonietta Coppola, Marica Rubino, Antonella Riva, Pasquale Striano   +3 more
wiley   +1 more source

Guanine base modifications in antisense oligonucleotides mitigate acute central nervous system toxicity. [PDF]

RSC Chem Biol
Katsuyama M, Matsubayashi T, Ying Y, Mon SSL, Kuroda T, Yoshida-Tanaka K, Hara RI, Yamada T, Ui-Tei K, Yoshioka K.   +9 more
europepmc   +1 more source

Precision therapies for genetic epilepsies in 2025: Promises and pitfalls

Epilepsia Open, EarlyView.
Abstract By targeting the underlying etiology, precision therapies offer an exciting paradigm shift to improve the stagnant outcomes of drug‐resistant epilepsies, including developmental and epileptic encephalopathies. Unlike conventional antiseizure medications (ASMs) which only treat the symptoms (seizures) but have no effect on the underlying ...
Shuyu Wang, Emilio Perucca, Samuel F. Berkovic, Piero Perucca   +3 more
wiley   +1 more source

Phenotype‐guided etiologic workup in a prospective cohort of 144 adults with developmental and epileptic encephalopathy

Epilepsia Open, EarlyView.
Abstract Objectives Adults with developmental and epileptic encephalopathies (DEEs) often enter adult neurology care without etiologic clarification because of incomplete transition from pediatric services, outdated investigations, and attenuation of childhood electro‐clinical features over time.
Giuseppe d’Orsi, Umberto Costantino, Maria Teresa Di Claudio, Carmela Pia Ferro, Pietro Palumbo, Mario Benvenuto, Massimiliano Copetti, Leonardo D'Agruma, Massimo Carella, Marco Castori, Orazio Palumbo   +10 more
wiley   +1 more source

Characterisation of Antisense Oligonucleotides by Ion-Pair Reversed-Phase UHPLC-HRMS: Method development using Design of Experiments. [PDF]

J Mass Spectrom
Triolo A, Tavani F, Barnini P, Furlanetto S, Orlandini S.   +4 more
europepmc   +1 more source

An Ocular Surface‐Targeting Nucleic Acid Hydrogel for Efficient Dry Eye Disease Treatment

Exploration, EarlyView.
A self‐assembled nucleic acid hydrogel modified with mucin‐1 (MUC1) aptamer is developed, which can actively target the mucin layer on the ocular surface and significantly extend its retention time after distillation. Inside the hydrogel, therapeutic siRNA is embedded and can effectively silence the inflammation gene expression, subsequently restoring ...
Yuhe Liu, Zimeng Zhai, Yangyang Huang, Fujun Wang, Zifei Wang, Lijuan Zhu, Ka Jiang, Xujiao Zhou, Xingtao Zhou, Ying Jie, Xiuming Jin, Chuan Zhang, Jiaxu Hong   +12 more
wiley   +1 more source

Chemically inducible antisense oligonucleotides for cell-specific gene silencing. [PDF]

RSC Chem Biol
Xun Z, Hai Y, Tang LJ, Jiang JH, Wu Z.
europepmc   +1 more source