Results 71 to 80 of about 109,213 (308)

Clinical advances of RNA therapeutics for treatment of neurological and neuromuscular diseases

RNA Biology, 2022
RNA therapeutics comprise a diverse group of oligonucleotide-based drugs such as antisense oligonucleotides (ASOs), small interfering RNAs (siRNAs), and short hairpin RNAs (shRNAs) that can be designed to selectively interact with drug targets currently ...
Anja Holm, Stine N. Hansen, Henrik Klitgaard, Sakari Kauppinen   +3 more
doaj   +1 more source

The endocannabinoid/cannabinoid receptor 2 system protects against cisplatin-induced hearing loss [PDF]

, 2018
Previous studies have demonstrated the presence of cannabinoid 2 receptor (CB2R) in the rat cochlea which was induced by cisplatin. In an organ of Corti-derived cell culture model, it was also shown that an agonist of the CB2R protected these cells ...
Asmita Dhukhwa, Debashree Mukherjea, Kelly Sheehan, Leonard P. Rybak, Leonard P. Rybak, Sandeep Sheth, Sumana Ghosh, Vickram Ramkumar, Vikrant Borse   +8 more
core   +4 more sources

Exploratory Analysis of ELP1 Expression in Whole Blood From Patients With Familial Dysautonomia

Annals of Clinical and Translational Neurology, EarlyView.
ABSTRACT Background Familial dysautonomia (FD) is a hereditary neurodevelopmental disorder caused by aberrant splicing of the ELP1 gene, leading to a tissue‐specific reduction in ELP1 protein expression. Preclinical models indicate that increasing ELP1 levels can mitigate disease manifestations.
Alejandra González‐Duarte, Lucy Norcliffe‐Kaufmann, Maria Luisa Cotrina, Zenith Khan, Kaia Dalamo, Patricio Millar Vernetti, Matthew Lawless, Elisabetta Morini, Monica Salani, Marla Weetall, Jana Narasimhan, Agostino G. Rocha, Susan A. Slaugenhaupt, Horacio Kaufmann   +13 more
wiley   +1 more source

The Use of Tricyclo-DNA Oligomers for the Treatment of Genetic Disorders

Biomedicines, 2017
Antisense Oligonucleotides (ASOs) represent very attractive therapeutic compounds for the treatment of numerous diseases. The antisense field has remarkably progressed over the last few years with the approval of the first antisense drugs and with ...
Philippine Aupy, Lucía Echevarría, Karima Relizani, Aurélie Goyenvalle   +3 more
doaj   +1 more source

Cloning, expression, and localization of a rat brain high-affinity glycine transporter [PDF]

, 1992
A cDNA clone encoding a glycine transporter has been isolated from rat brain by a combined PCR and plaque-hybridization strategy. mRNA synthesized from this clone (designated GLYT1) directs the expression of sodium-and chloride-dependent, high-affinity ...
Brecha, Nicholas, Davidson, Norman, Guastella, John, Lester, Henry A., Weigmann, Christine   +4 more
core   +1 more source

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

Identification of Novel Antisense-Mediated Exon Skipping Targets in DYSF for Therapeutic Treatment of Dysferlinopathy

Molecular Therapy: Nucleic Acids, 2018
Dysferlinopathy is a progressive myopathy caused by mutations in the dysferlin (DYSF) gene. Dysferlin protein plays a major role in plasma-membrane resealing.
Joshua J.A. Lee, Rika Maruyama, William Duddy, Hidetoshi Sakurai, Toshifumi Yokota   +4 more
doaj   +1 more source

Potentiation of thrombus instability: a contributory mechanism to the effectiveness of antithrombotic medications [PDF]

, 2018
© The Author(s) 2018The stability of an arterial thrombus, determined by its structure and ability to resist endogenous fibrinolysis, is a major determinant of the extent of infarction that results from coronary or cerebrovascular thrombosis.
A Gast, AD Michelson, B Labarthe, B Shen, C Patrono, CT Ammollo, D Capodanno, D Gailani, DA Gorog, Diana A. Gorog, DM Coenen, E Álvarez, ER Bates, F Kabirian, F Müller, F Semeraro, G Stephens, H Brogren, HE Speich, HE Speich, HJ Weiss, HJ Weiss, HR Baumgartner, J Zilberman-Rudenko, JD McFadyen, JI Weitz, JM Cosemans, JP Collet, JP Collet, K Hosokawa, K Nagatsuka, KS Sakariassen, L Valerio, LF Brass, M Cattaneo, M Farag, M Humbert, M Larsson, M Li, M Ungerer, MA Bailey, MI Furman, MJ Price, ML Montfoort von, MS Sabatine, P Andre, P Seizer, PA Borne von dem, PP Wadowski, R Hellmuth, R Nergiz-Unal, RA Ajjan, S Goto, S Goto, SA Shaya, SB Larsen, SJ Marciniak, T Heitzer, T Renné, VT Turitto, VT Turitto, WA Schumacher, WS Nesbitt, X Wang, Y Yano, YC Lau, ZM Ruggeri   +66 more
core   +2 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

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