Results 61 to 70 of about 83,454 (234)

Functional and Structural Evidence of Neurofluid Circuit Aberrations in Huntington Disease

Annals of Clinical and Translational Neurology, EarlyView.
ABSTRACT Objective Disrupted neurofluid regulation may contribute to neurodegeneration in Huntington disease (HD). Because neurofluid pathways influence waste clearance, inflammation, and the distribution of central nervous system (CNS)–delivered therapeutics, understanding their dysfunction is increasingly important as targeted treatments emerge.
Kilian Hett, Abigail Dubois, Melanie Leguizamon, Alexander Song, Paula Trujillo, Colin D. McKnight, Ciaran M. Considine, Manus J. Donahue, Daniel O. Claassen   +8 more
wiley   +1 more source

Cyclodextrin-Based Nanoparticles for Delivery of Antisense Oligonucleotides Targeting Huntingtin

Pharmaceutics, 2023
Huntington’s disease (HD) is a progressive inherited neurodegenerative disease caused by a CAG repeat expansion in the huntingtin gene, which is translated into the pathologic mutant huntingtin (mHTT) protein.
Monique C. P. Mendonça, Yao Sun, Michael F. Cronin, Andrew J. Lindsay, John F. Cryan, Caitriona M. O’Driscoll   +5 more
doaj   +1 more source

Targeted knock-down of miR21 primary transcripts using snoMEN vectors induces apoptosis in human cancer cell lines [PDF]

, 2015
We have previously reported an antisense technology, 'snoMEN vectors', for targeted knock-down of protein coding mRNAs using human snoRNAs manipulated to contain short regions of sequence complementarity with the mRNA target. Here we characterise the use
A Kozomara, A Rodriguez, AG Matera, AK Leung, AM Burroughs, AM Cheng, Angus I. Lamond, B Bai, C Ender, Christophe Antoniewski, CL Ahlenstiel, D De Paula, D Iliopoulos, Dalila Bensaddek, EC Lai, EC Lai, EJ Chapman, EJ Jung, Fabio Avolio, FJ Sheedy, FM Boisvert, FM Boisvert, G Reimer, GS Pall, I Rebbapragada, J Elmen, J Krutzfeldt, J Liu, J Weischenfeldt, JC Politz, JS Andersen, JS Andersen, K Yamada, Kayo Yamada, KK Farh, L Agranat, L Trinkle-Mulcahy, LB Frankel, LB Weinstein, M Negrini, M Ono, M Ono, M Ono, M Scherr, ME Hatley, Motoharu Ono, MS Scott, MS Scott, N Bushati, O Isken, RS Coffin, S Baskerville, S Griffiths-Jones, SD Selcuklu, T Kiss, Vackar Afzal, Y Ahmad, YK Kim, YS Lee, YW Lam   +59 more
core   +5 more sources

Expanding Chemical Space of Nucleic Acid Nanoparticles for Tunable Antiviral‐Like Immunomodulatory Responses and Potent Adjuvant Activity

Advanced Functional Materials, EarlyView.
We introduce a nucleic acid nanoparticle (NANP) platform designed to be rrecognized by the human innate immune system in a regulated manner. By changing chemical composition while maintaining constant architectural parameters, we identify key determinants of immunorecognition enabling the rational design of NANPs with tunable immune activation profiles
Martin Panigaj, Hannah S. Newton, Jian Wang, Laxmi Pandey, Yelixza I. Avilla, Phong Nguyen, Morgan Chandler, Justin Halman, Yasmine Radwan, Stephanie Smith, Elijah Edmondson, Simone Difilippantonio, Chelsea Sanders, Bao Tran, Yongmei Zhao, Shaojun Xie, Edward Cedrone, Barry W. Neun, Meni Wanunu, Nikolay V. Dokholyan, Marina A. Dobrovolskaia, Kirill A. Afonin   +21 more
wiley   +1 more source

Role of noncoding RNA in vascular remodelling [PDF]

, 2016
Purpose of review: Noncoding RNAs (ncRNAs), such as microRNAs (miRNAs) and long noncoding RNAs (lncRNAs) are becoming fundamentally important in the pathophysiology relating to injury-induced vascular remodelling.
Baker, Andrew H., Bradshaw, Angela C., Deng, Lin   +2 more
core   +1 more source

Delivery of Antisense Oligonucleotides to the Mouse Retina [PDF]

, 2022
AbstractThe eye is the organ in charge of vision and, given its properties, has become an excellent organ to test genetic therapies, including antisense oligonucleotide (AON) technology. In fact, the first AON receiving FDA and EMA approval was meant to treat an eye condition.
openaire   +3 more sources

RNA‐Micelles as Self‐Assembling Structures for Efficient Co‐Delivery of Synergistic siRNA and Nucleoside Analogues to Treat CRC Lung Metastasis

Advanced Functional Materials, EarlyView.
Two kinds of self‐assembled RNA micelles were used to co‐deliver synergistic siRNA and nucleoside analogues for the treatment of colorectal cancer lung metastases. Near‐complete elimination of lung cancer metastases was confirmed in an orthotopic lung metastasis model constructed using human colorectal cancer lung metastases patient surgical samples to
Kai Jin, Piotr Rychahou, Daniel W. Binzel, B. Mark Evers, Peixuan Guo   +4 more
wiley   +1 more source

Calcium-Enhanced Medium-Based Delivery of Splice Modulating Antisense Oligonucleotides in 2D and 3D hiPSC-Derived Neuronal Models

Biomedicines
Antisense technology demonstrates significant potential for addressing inherited brain diseases, with over a dozen products already available and numerous others in the development pipeline.
Ronald A. M. Buijsen, Linda M. van der Graaf, Elsa C. Kuijper, Barry A. Pepers, Elena Daoutsali, Lotte Weel, Vered Raz, David A. Parfitt, Willeke M. C. van Roon-Mom   +8 more
doaj   +1 more source

Nanodiamond as a vector for siRNA delivery to Ewing sarcoma cells

, 2011
We investigated the ability of diamond nanoparticles (nanodiamonds, NDs) to deliver small interfering RNA (siRNA) in Ewing sarcoma cells, in the perspective of in vivo anti-cancer nucleic acid drug delivery.
Barnard, Bertrand, Bolte, Boudou, Caliceti, Chang, Chen, Choosakoonkriang, Chow, Chung, Creusat, Dantelle, de Martimprey, Diaz-Lopez, El Badawy, Elhamess, Faklaris, Fattal, Guan, Horcajada, Huang, Huang, Kaiser, Kurosaki, Liang, Liu, Maksimenko, Maksimenko, Mohan, Nakada, Pouton, Qi, Ramon, Rondin, Schrand, Schrand, Simovic, Tanaka, Tang, Toub, Vaijayanthimala, Vial, Xia, Xu, Yuan, Yuan, Zhang, Zoubek   +47 more
core   +3 more sources

VIVID: A qPCR‐Based Platform for Sensitive and Quantitative In Vivo Tracking of Extracellular Vesicles

Advanced Functional Materials, EarlyView.
This study introduces VIVID (Vesicle In Vivo Identification using DNA), a qPCR‐based platform that tracks PCR‐amplifiable DNA tags loaded in the EVs for accurate and quantifiable EV biodistribution in vivo. ABSTRACT Extracellular vesicles (EVs) represent promising carriers for nucleic acid therapeutics, offering advantages over synthetic nanoparticles ...
Oscar Boyadjian, Talin Ebrahimian, Jonathan Miranda O'Connor, Janusz Rak, Stephanie Lehoux, Maryam Tabrizian   +5 more
wiley   +1 more source