Results 131 to 140 of about 171,175 (241)

Silencing with precision: In silico strategies for antisense oligonucleotide engineering. [PDF]

Mol Biol Rep
Nagaraj A, Kavana CP, Harendra B, Srinivasa S, Dharmashekara C, Kollur SP, Shreevatsa B, Shivamallu C.   +7 more
europepmc   +1 more source

A prospective natural history study protocol for clinical trial readiness in synaptic disorders

Epilepsia, EarlyView.
Abstract Objective STXBP1‐related disorder (STXBP1‐RD) and SYNGAP1‐related disorder (SYNGAP1‐RD) are two common genetic synaptopathies that are associated with epilepsy, developmental delay, intellectual developmental disorder, and behavioral problems.
Jillian L. McKee, Sarah M. Ruggiero, Kristin Cunningham, JoeyLynn Coyne, Ian McSalley, Michael C. Kaufman, Bintou Bane, Torrey Chisari, Jonathan Toib, Carlyn Glatts, Sarah Tefft, Julie M. Orlando, Viveknarayanan Padmanabhan, Alexander K. Gonzalez, Alicia Harrison, Charlene Woo, Stephanie A. Zbikowski, Rency Dhaduk, Johanna Mercurio, Macie McCarthy, Jan H. Magielski, Zachary Grinspan, Megan Abbott, Juliet Knowles, Hsiao‐Tuan Chao, Katherine Xiong, Elizabeth Berry‐Kravis, Sepideh Tabarestani, J. Michael Graglia, Kathryn Helde, Virginie McNamar, Charlene Son Rigby, James Goss, Scott Demarest, Andrea Miele, Benjamin Prosser, Michael J. Boland, Samuel R. Pierce, Ingo Helbig   +38 more
wiley   +1 more source

Tripled-Stranded Antisense Oligonucleotide for Biomarker-Activated Suppression of Essential Genes. [PDF]

Chembiochem
Drozd VS, Tafran L, Zaramenskih NV, Panova YA, Hussein Z, Rybalko DS, Kolpashchikov DM, Eldeeb AA.   +7 more
europepmc   +1 more source

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

Imaging-Assisted Antisense Oligonucleotide Delivery for Tumor-Targeted Gene Therapy

Antisense oligonucleotide (ASO) represents a class of practical tools for targeting undruggable oncogenes with several candidates currently undergoing clinical investigation. The advancement of antisense therapeutics necessitates comprehensive approaches
Hanwen Liao (18396321), Siyi Wang (1412581), Yan Zhang (8098), David Zixiang Dai (18396324), Jinbo Li (1333470), Chenghong Zhu (16631427), Xiaoning Wang (16150)   +6 more
core   +1 more source

Clinical Pharmacology of Eplontersen, the First Approved GalNAc-Conjugated Antisense Oligonucleotide. [PDF]

J Clin Pharmacol
Yu RZ, Diep JK, Gao X, Arfvidsson C, Quartino A.   +4 more
europepmc   +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

Advances in antisense oligonucleotide treatment for cancer. [PDF]

Jpn J Clin Oncol
Zhu L, Muhtar P, Goyama S, Yoshimi A.
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

Antisense oligonucleotide-mediated disruption of mRNA localisation inhibits angiogenesis. [PDF]

Angiogenesis
Augustine J, Owuamalam PO, Nicell J, Barabas P, Chatterjee S, O'Hare M, Curtis TM, Costa G.   +7 more
europepmc   +1 more source