Antisense Oligonucleotide-Mediated Transcript Knockdown in Zebrafish. [PDF]
PLoS ONE, 2015 Antisense oligonucleotides (ASOs) are synthetic, single-strand RNA-DNA hybrids that induce catalytic degradation of complementary cellular RNAs via RNase H.
Andrea Pauli +4 more
doaj +1 more source
Identification of antisense nucleic acid hybridization sites in mRNA molecules with self-quenching fluorescent reporter molecules [PDF]
, 2005 We describe a physical mRNA mapping strategy employing fluorescent self-quenching reporter molecules (SQRMs) that facilitates the identification of mRNA sequence accessible for hybridization with antisense nucleic acids in vitro and in vivo, real time ...
Do, Bao T. +11 more
core +2 more sources
Peroxisome Proliferator-Activated Receptor alpha (PPAR alpha) down-regulation in cystic fibrosis lymphocytes [PDF]
, 2006 Background: PPARs exhibit anti-inflammatory capacities and are potential modulators of the inflammatory response. We hypothesized that their expression and/or function may be altered in cystic fibrosis (CF), a disorder characterized by an excessive host ...
A Augarten +53 more
core +4 more sources
Nusinersen: A Novel Antisense Oligonucleotide for the Treatment of Spinal Muscular Atrophy.
The Journal of Pediatric Pharmacology and Therapeutics, 2019 Spinal muscular atrophy (SMA) encompasses a group of autosomal recessively inherited degenerative neuromuscular disorders. They range in severity from neonatal onset with rapidly progressive weakness and early mortality (SMA-1), to onset in infancy (SMA ...
E. Neil, E. Bisaccia
semanticscholar +1 more source
Investigating synthetic oligonucleotide targeting of miR31 in Duchenne muscular dystrophy [PDF]
, 2016 Exon-skipping via synthetic antisense oligonucleotides represents one of the most promising potential therapies for Duchenne muscular dystrophy (DMD), yet this approach is highly sequence-specific and thus each oligonucleotide is of benefit to only a ...
Hildyard, J C W, Wells, D J
core +1 more source
Nonviral delivery systems for antisense oligonucleotide therapeutics
Biomaterials Research, 2022 Antisense oligonucleotides (ASOs) are an important tool for the treatment of many genetic disorders. However, similar to other gene drugs, vectors are often required to protect them from degradation and clearance, and to accomplish their transport in ...
Si Huang +5 more
doaj +1 more source
Role of G{alpha}12 and G{alpha}13 as Novel Switches for the Activity of Nrf2, a Key Antioxidative Transcription Factor [PDF]
, 2007 G{alpha}12 and G{alpha}13 function as molecular regulators responding to extracellular stimuli. NF-E2-related factor 2 (Nrf2) is involved in a protective adaptive response to oxidative stress.
Cho, Min Kyung +6 more
core +2 more sources
Antisense Oligonucleotide: Basic Concepts and Therapeutic Application in Inflammatory Bowel Disease
Frontiers in Pharmacology, 2019 Several molecular technologies aimed at regulating gene expression that have been recently developed as a strategy to combat inflammatory and neoplastic diseases.
D. Di Fusco +5 more
semanticscholar +1 more source
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 +8 more
doaj
Targeting several CAG expansion diseases by a single antisense oligonucleotide. [PDF]
PLoS ONE, 2011 To date there are 9 known diseases caused by an expanded polyglutamine repeat, with the most prevalent being Huntington's disease. Huntington's disease is a progressive autosomal dominant neurodegenerative disorder for which currently no therapy is ...
Melvin M Evers +7 more
doaj +1 more source