Results 11 to 20 of about 443,792 (339)

Antisense oligonucleotides [PDF]

Neurology Genetics, 2019
There are few disease-modifying therapeutics for neurodegenerative diseases, but successes on the development of antisense oligonucleotide (ASO) therapeutics for spinal muscular atrophy and Duchenne muscular dystrophy predict a robust future for ASOs in medicine.
Scoles, Daniel R., Minikel, Eric V., Pulst, Stefan M.   +2 more
openaire   +3 more sources

Trial of Antisense Oligonucleotide Tofersen for SOD1 ALS.

New England Journal of Medicine, 2022
BACKGROUND The intrathecally administered antisense oligonucleotide tofersen reduces synthesis of the superoxide dismutase 1 (SOD1) protein and is being studied in patients with amyotrophic lateral sclerosis (ALS) associated with mutations in SOD1 (SOD1 ...
Timothy A. Miller, M. Cudkowicz, A. Genge, P. Shaw, G. Sobue, R. Bucelli, A. Chiò, P. van Damme, A. Ludolph, J. Glass, Jinsy A. Andrews, Suma Babu, M. Benatar, C. Mcdermott, T. Cochrane, Sowmya Chary, Sheena Chew, Handong Zhu, Fan Wu, I. Nestorov, D. Graham, P. Sun, M. McNeill, L. Fanning, T. Ferguson, S. Fradette   +25 more
semanticscholar   +1 more source

Antisense technology: A review

Journal of Biological Chemistry, 2021
Antisense technology is beginning to deliver on the broad promise of the technology. Ten RNA-targeted drugs including eight single-strand antisense drugs (ASOs) and two double-strand ASOs (siRNAs) have now been approved for commercial use, and the ASOs ...
S. Crooke, Xue-hai Liang, B. Baker, R. Crooke   +3 more
semanticscholar   +1 more source

Antisense Oligonucleotides: An Emerging Area in Drug Discovery and Development

Journal of Clinical Medicine, 2020
Antisense oligonucleotides (ASOs) bind sequence specifically to the target RNA and modulate protein expression through several different mechanisms. The ASO field is an emerging area of drug development that targets the disease source at the RNA level ...
Karishma Dhuri, C. Bechtold, Elias Quijano, H. Pham, Anisha Gupta, A. Vikram, R. Bahal   +6 more
semanticscholar   +1 more source

RNA-Based Therapeutics: From Antisense Oligonucleotides to miRNAs

Cells, 2020
The first therapeutic nucleic acid, a DNA oligonucleotide, was approved for clinical use in 1998. Twenty years later, in 2018, the first therapeutic RNA-based oligonucleotide was United States Food and Drug Administration (FDA) approved. This promises to
Sarah Bajan, G. Hutvagner
semanticscholar   +1 more source

Genetic Modification of KNAT7 Transcription Factor Expression Enhances Saccharification and Reduces Recalcitrance of Woody Biomass in Poplars

Frontiers in Plant Science, 2021
The precise role of KNAT7 transcription factors (TFs) in regulating secondary cell wall (SCW) biosynthesis in poplars has remained unknown, while our understanding of KNAT7 functions in other plants is continuously evolving.
Yogesh Kumar Ahlawat, Yogesh Kumar Ahlawat, Akula Nookaraju, Anne E. Harman-Ware, Crissa Doeppke, Ajaya K. Biswal, Ajaya K. Biswal, Chandrashekhar P. Joshi   +7 more
doaj   +1 more source

Translation of mRNA injected into Xenopus oocytes is specifically inhibited by antisense RNA. [PDF]

, 1985
The bacteriophage SP6 promoter and RNA polymerase were used to synthesize sense and antisense RNAs coding for the enzymes thymidine kinase (TK) and chloramphenicol acetyl transferase (CAT).
HARLAND, Richard M., Weintraub, H
core   +2 more sources

Antisense oligonucleotide modulation of non-productive alternative splicing upregulates gene expression

Nature Communications, 2020
While most monogenic diseases are caused by loss or reduction of protein function, the need for technologies that can selectively increase levels of protein in native tissues remains.
K. Lim, Zhou Han, H. Y. Jeon, Jacob Kach, Enxuan Jing, Sebastien M. Weyn-Vanhentenryck, Mikaela Downs, Anna Corrionero, Raymond Oh, Juergen Scharner, Aditya Venkatesh, Sophina Ji, G. Liau, B. Ticho, H. Nash, I. Aznarez   +15 more
semanticscholar   +1 more source

Correction to the pathogenic alternative splicing, caused by the common GNB3 c.825C>T allele, using a novel, antisense morpholino [PDF]

, 2016
The very common GNB3 c.825C>T polymorphism (rs5443), is present in approximately half of all human chromosomes. Significantly the presence of the GNB3 825T allele has been strongly associated, with predisposition to essential hypertension ...
Lester, Douglas H., McGlinchey, Jonathan C. P., Tummala, Hemanth   +2 more
core   +3 more sources

An apolipoprotein B antisense oligonucleotide lowers LDL cholesterol in hyperlipidemic mice without causing hepatic steatosis

Journal of Lipid Research, 2005
High levels of plasma apolipoprotein B-100 (apoB-100), the principal apolipoprotein of LDL, are associated with cardiovascular disease. We hypothesized that suppression of apoB-100 mRNA by an antisense oligonucleotide (ASO) would reduce LDL cholesterol ...
Rosanne M. Crooke, Mark J. Graham, Kristina M. Lemonidis, Charles P. Whipple, Seonjoon Koo, Ranjan J. Perera   +5 more
doaj   +1 more source