Results 11 to 20 of about 120,043 (332)
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.
Eric Vallabh Minikel +2 more
openaire +3 more sources
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 +9 more
doaj +1 more source
The potential of antisense oligonucleotide therapies for inherited childhood lung diseases [PDF]
, 2018 Antisense oligonucleotides are an emerging therapeutic option to treat diseases with known genetic origin. In the age of personalised medicines, antisense oligonucleotides can sometimes be designed to target and bypass or overcome a patient’s genetic ...
Fletcher, S. +6 more
core +5 more sources
In Vivo Models for the Evaluation of Antisense Oligonucleotides in Skin [PDF]
, 2022 Here, we describe an in vivo model in which antisense oligonucleotides were preclinically evaluated in reconstituted patient and healthy control skin.
Bremer, Jeroen, van den Akker, Peter C
core +1 more source
Encapsulation of DNA in negatively charged liposomes and inhibition of bacterial gene expression with fluid liposome-encapsulated antisense oligonucleotides [PDF]
, 2001 Antisense therapy for the treatment of bacterial infections is a very attractive alternative to overcome drug resistance problems. However, the penetration of antisense oligonucleotides into bacterial cells is a major huddle that has delayed research and
Desjardins, Annie +3 more
core +1 more source
Beilstein Journal of Organic Chemistry, 2021 Chemical modifications have been extensively used for therapeutic oligonucleotides because they strongly enhance the stability against nucleases, binding affinity to the targets, and efficacy. We previously reported that oligonucleotides modified with an
Naohiro Horie +3 more
doaj +1 more source
Nucleobase-modified antisense oligonucleotides containing 5-(phenyltriazol)-2′-deoxyuridine nucleotides induce exon-skipping:In vitro [PDF]
, 2017 We investigated the potential of nucleobase-modified antisense oligonucleotides to induce exon-skipping, and found that 5-(phenyltriazol)-2′-deoxyuridine-modified antisense oligonucleotides induced efficient exon-skipping in vitro.
Hornum, Mick +4 more
core +1 more source
Expression of ssDNA in Mammalian Cells
BioTechniques, 2003 Antisense therapy involves the use of antisense oligonucleotides for altering targeted gene function. However, the low efficiency of cell delivery of antisense oligonucleotides has limited the efficacy of antisense therapeutic approaches.
Yin Chen, Yong-Jie Ji, Charles Conrad
doaj +1 more source
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 +4 more
doaj +1 more source
Polymerase-endonuclease amplification reaction (PEAR) for large-scale enzymatic production of antisense oligonucleotides. [PDF]
PLoS ONE, 2010 Antisense oligonucleotides targeting microRNAs or their mRNA targets prove to be powerful tools for molecular biology research and may eventually emerge as new therapeutic agents.
Xiaolong Wang +2 more
doaj +1 more source