Results 1 to 10 of about 270,275 (243)

Antisense Oligonucleotide-Based Therapy of Viral Infections

Pharmaceutics, 2021
Nucleic acid-based therapeutics have demonstrated their efficacy in the treatment of various diseases and vaccine development. Antisense oligonucleotide (ASO) technology exploits a single-strand short oligonucleotide to either cause target RNA ...
Li-Min Huang
exaly   +5 more sources

ASOG: AntiSense Oligonucleotide Generator [PDF]

Computational and Structural Biotechnology Journal
Antisense oligonucleotides (ASOs) are used in both fundamental research and clinical applications to modulate gene expression by targeting the RNA transcript of specific genes.
Jonah Kimi, Patricia Korczak, Brune Vialet, Eric Roubin, Philippe Barthélémy, Sébastien Campagne, Florian Malard   +6 more
doaj   +2 more sources

Phase 1–2 Trial of Antisense Oligonucleotide Tofersen for SOD1 ALS

New England Journal of Medicine, 2020
BACKGROUND Tofersen is an antisense oligonucleotide that mediates the degradation of superoxide dismutase 1 (SOD1) messenger RNA to reduce SOD1 protein synthesis.
Timothy M Miller, Merit E Cudkowicz, Pamela J Shaw   +2 more
exaly   +2 more sources

NS-065/NCNP-01: An Antisense Oligonucleotide for Potential Treatment of Exon 53 Skipping in Duchenne Muscular Dystrophy

Molecular Therapy: Nucleic Acids, 2018
Duchenne muscular dystrophy (DMD), the most common lethal heritable childhood disease, is caused by mutations in the DMD gene that result in the absence of functional dystrophin protein.
Naoki Watanabe, Tetsuya Nagata, Youhei Satou, Satoru Masuda, Takashi Saito, Hidetoshi Kitagawa, Hirofumi Komaki, Kazuchika Takagaki, Shin’ichi Takeda   +8 more
doaj   +2 more sources

Antisense oligonucleotide therapy for KCNT1 encephalopathy

JCI Insight, 2022
Developmental and epileptic encephalopathies (DEEs) are characterized by pharmaco-resistant seizures with concomitant intellectual disability. Epilepsy of infancy with migrating focal seizures (EIMFS) is one of the most severe of these syndromes. De novo
Lisseth Estefania Burbano, Melody Li, Nikola Jancovski, Paymaan Jafar-Nejad, Kay Richards, Alicia Sedo, Armand Soriano, Ben Rollo, Linghan Jia, Elena V. Gazina, Sandra Piltz, Fatwa Adikusuma, Paul Q. Thomas, Helen Kopsidas, Frank Rigo, Christopher A. Reid, Snezana Maljevic, Steven Petrou   +17 more
doaj   +2 more sources

Potential antisense oligonucleotide targeting LINC00673 on proliferation and apoptosis in tongue squamous cell carcinoma [PDF]

Journal of Taibah University Medical Sciences
الملخص: أهداف البحث: تزداد معدلات الإصابة بسرطان اللسان، المعروف بطبيعته العدوانية، سنويًّا في إندونيسيا وعلى مستوى العالم. ويرتبط أحد العوامل الجزيئية المرتبطة بسرطان اللسان البشري بارتفاع التعبير عن الحمض النووي الريبوزي الطويل غير المشفِّر (LINC٠٠٦٧٣)
Kadek Gede Putra Wibawa, DDS, Supriatno, PhD, Juni Handajani, PhD   +2 more
doaj   +2 more sources

Antisense Oligonucleotide-Mediated Removal of the Polyglutamine Repeat in Spinocerebellar Ataxia Type 3 Mice

Molecular Therapy: Nucleic Acids, 2017
Spinocerebellar ataxia type 3 (SCA3) is a currently incurable neurodegenerative disorder caused by a CAG triplet expansion in exon 10 of the ATXN3 gene.
Lodewijk J.A. Toonen, Frank Rigo, Haico van Attikum, Willeke M.C. van Roon-Mom   +3 more
doaj   +2 more sources

Angubindin-1 opens the blood–brain barrier in vivo for delivery of antisense oligonucleotide to the central nervous system

Journal of Controlled Release, 2018
&NA; Within the field of RNA therapeutics, antisense oligonucleotide‐based therapeutics are a potentially powerful means of treating intractable diseases.
Hiroya Kuwahara, Masuo Kondoh, Kie Yoshida-Tanaka   +2 more
exaly   +2 more sources

Antisense Oligonucleotide-Based Therapy for Neuromuscular Disease

Molecules, 2017
Neuromuscular disorders such as Duchenne Muscular Dystrophy and Spinal Muscular Atrophy are neurodegenerative genetic diseases characterized primarily by muscle weakness and wasting.
Valentina Sardone, Haiyan Zhou, Francesco Muntoni, Alessandra Ferlini, Maria Sofia Falzarano   +4 more
doaj   +2 more sources

An Investigation into the Potential of Targeting Escherichia coli rne mRNA with Locked Nucleic Acid (LNA) Gapmers as an Antibacterial Strategy

Molecules, 2021
The increase in antibacterial resistance is a serious challenge for both the health and defence sectors and there is a need for both novel antibacterial targets and antibacterial strategies.
Layla R. Goddard, Charlotte E. Mardle, Hassan Gneid, Ciara G. Ball, Darren M. Gowers, Helen S. Atkins, Louise E. Butt, Jonathan K. Watts, Helen A. Vincent, Anastasia J. Callaghan   +9 more
doaj   +1 more source