Results 131 to 140 of about 125,197 (343)

Self‐Immolative Gels: Programmable Degradation Using Self‐Immolative Linkers and Polymers

Chemistry – A European Journal, EarlyView.
Self‐immolative linkers and polymers undergo bond‐cleaving reaction cascades in response to specific stimuli. Their incorporation into gels can be harnessed to release cargo, induce property changes, or trigger degradation in a highly controlled manner. These functions can be employed in applications such as sensing and drug delivery.
Chuanfeng Li, Elizabeth R. Gillies
wiley   +1 more source

Correction of Aberrant Splicing of the Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) Gene by Antisense Oligonucleotides [PDF]

, 1999
Kenneth J. Friedman, Jolanta Kole, Jonathan Cohn, Michael R. Knowles, Lawrence M. Silverman, Ryszard Kole   +5 more
openalex   +1 more source

Role of SoxE transcription factors in development and disease

Developmental Dynamics, EarlyView.
Abstract Sox8, Sox9, and Sox10 arose by multiple rounds of genome duplications from a single SoxE gene in ancestral vertebrates. In this review, we will briefly discuss the molecular structure and function of SoxE transcription factors and their evolutionary origin. We will then discuss their expression, function, and developmental disorders.
Merin Lawrence, Gerhard Schlosser
wiley   +1 more source

Antisense oligonucleotides to proliferating cell nuclear antigen and Ki-67 inhibit human mesangial cell proliferation. [PDF]

, 1996
Yohei Maeshima, Naoki Kashihara, Hitoshi Sugiyama, Hirofumi Makino, Zensuke Ota   +4 more
openalex   +1 more source

Antisense oligonucleotides targeting the miR-29b binding site in the GRN mRNA increase progranulin translation [PDF]

, 2022
Geetika Aggarwal, Subhashis Banerjee, Spencer A. Jones, Yousri Benchaar, Jasmine Bélanger, Myriam Sévigny, Denise M. Smith, Michael L. Niehoff, Monica Pavlack, Ian Mitchelle S. de Vera, Terri L. Petkau, Blair R. Leavitt, Karen Ling, Paymaan Jafar‐Nejad, Frank Rigo, John E. Morley, Susan A. Farr, Paul A. Dutchak, Chantelle F. Sephton, Andrew D. Nguyen   +19 more
openalex   +1 more source

Evolution of the European Medicines Agency clinical guidelines for epilepsy drug development between 2010 and 2025: A comparative analysis by the ILAE Task Force on Regulatory Affairs

Epilepsia, EarlyView.
Abstract Objective The latest European Medicines Agency (EMA) guideline on the clinical investigation of medicines to treat epileptic disorders was adopted by the EMA Committee for Medicinal Products for Human Use in 2025. We compared this guideline with the previous version (2010), highlighting areas where significant revisions were introduced ...
Stéphane Auvin, Alexis Arzimanoglou, Jacqueline French, Kelly G. Knupp, Lieven Lagae, Eugen Trinka, Dennis Dlugos, Emilio Perucca   +7 more
wiley   +1 more source

Investigation of the activity of phosphothioate and phosphothioate-LNA-modified oligonucleotides against HIV-1

Frontiers in Medicine
This study investigated the antiretroviral efficacy, toxicity profile, and cellular uptake of phosphothioate (PS) and PS/LNA-modified oligonucleotides within an in vitro HIV infection model.
Ludmila Gotfrid, Kirill Elfimov, Maria Gashnikova, Aleksey Totmenin, Aleksandr Agaphonov, Natalya Gashnikova   +5 more
doaj   +1 more source

Neonatal developmental and epileptic encephalopathy with movement disorder and arthrogryposis: A shared phenotype across brain‐expressed sodium channelopathies

Epilepsia, EarlyView.
Abstract Objective Neonatal developmental and epileptic encephalopathy with movement disorder and arthrogryposis (NDEEMA) represents the most severe end of the gain‐of‐function (GOF) SCN1A disorder spectrum. Sporadic cases of congenital arthrogryposis have also been reported in individuals with SCN2A‐, SCN3A‐, and SCN8A‐related developmental and ...
Sopio Gverdtsiteli, Sebastian Ortiz, Tobias Brünger, Francesca Furia, Carmen Barba, Trine Bjørg‐Hammer, Ingo Borggraefe, Roberto Caraballo, Sebahattin Cirak, Alberto Espeche, Walid Fazeli, Renzo Guerrini, Matias Juanes, Karin Kassahn, Maria Kinali, Johannes Krämer, Judith Kröll, Maria Concepción Miranda Herrero, Renske Oegema, Katrin Ounap, Oscar Peñuela, Konrad Platzer, Asuri Narayan Prasad, Aurora Pujol, Karit Reinson, Alfonso Represa, Eugenia Roza, Gabriela Reyes Valenzuela, Agustí Rodríguez‐Palmero, Suzanne Sallevelt, Maria Iciar Sanchez‐Albiusa, Ingrid E. Scheffer, Cory Smid, Carl E. Stafstrom, Eva‐Lena Stattin, Jen R. Suarez, Steffen Syrbe, Kette D. Valente, Matias Wagner, Saskia Wortmann, Elena Gardella, Dennis Lal, Andreas Brunklaus, Rikke S. Møller   +43 more
wiley   +1 more source

Integrin Targeted Delivery of Gene Therapeutics

Theranostics, 2011
Integrins have become key targets for molecular imaging and for selective delivery of anti-cancer agents. Here we review recent work concerning the targeted delivery of antisense and siRNA oligonucleotides via integrins. A variety of approaches have been
Rudy L Juliano, Xin Ming, Osamu Nakagawa, Rongzuo Xu, Hoon Yoo
doaj  

An n‐of‐1 gene‐directed drug repurposing trial for an ultrarare genetic condition

Epilepsia, EarlyView.
Abstract Objective Gain‐of‐function (GoF) variants in the KCNC1 potassium channel subunit gene (Kv3.1) cause motor/cognitive delays and hypotonia and have been associated with seizures. Fluoxetine has inhibitory effects on Kv3.1. However, open‐label nonrandomized administration is insufficient to guide clinical decision‐making in ultrarare conditions ...
Vedika Jha, Christina Tsetsos, Mark Bedford, Gregory Costain, Sunita Vohra, Juan Pablo Diaz Martinez, Elise Heon, Jacob Vorstman, Carolina Gorodetsky, Ajoy Vincent, Jesiqua Rapley, Lori Anderson, Evdokia Anagnostou, Danielle A. Baribeau   +13 more
wiley   +1 more source