Results 21 to 30 of about 45,567 (253)
Molecular Therapy: Nucleic Acids, 2018 Antisense oligonucleotide (AON) therapy for Duchenne muscular dystrophy has drawn great attention in preclinical and clinical trials, but its therapeutic applications are still limited due to inefficient delivery.
Mingxing Wang +4 more
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International Journal of Nanomedicine, 2023 Marta Kutwin,1 Malwina Sosnowska,1 Agnieszka Ostrowska,1 Maciej Trzaskowski,2 Agata Lange,1 Mateusz Wierzbicki,1 Sławomir Jaworski1 1Department of Nanobiotechnology, Institute of Biology, Warsaw University of Life Sciences, Warsaw, 02-786, Poland;
Kutwin M +6 more
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Antisense oligonucleotide based therapeutics and its applications against bacterial infections
Medicine in Drug Discovery, 2023 The transcriptome of a cell governs the overall cellular expression of proteins, hence, targeting mRNA holds promise as a therapeutic strategy in the field of drug discovery.
Nupur Angrish, Garima Khare
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Drug Delivery, 2017 Antisense oligonucleotide therapy for Duchenne muscular dystrophy has shown great potential in preclinical and clinical trials, but its therapeutic applications are still limited due to inefficient delivery.
Mingxing Wang +4 more
doaj +1 more source
Delivery of Antisense Oligonucleotides to the Mouse Retina [PDF]
, 2022 AbstractThe eye is the organ in charge of vision and, given its properties, has become an excellent organ to test genetic therapies, including antisense oligonucleotide (AON) technology. In fact, the first AON receiving FDA and EMA approval was meant to treat an eye condition.
openaire +3 more sources
In Vitro Validation of Phosphorodiamidate Morpholino Oligomers
Molecules, 2019 One of the crucial aspects of screening antisense oligonucleotides destined for therapeutic application is confidence that the antisense oligomer is delivered efficiently into cultured cells.
May T. Aung-Htut +4 more
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Molecular Therapy: Nucleic Acids, 2012 Many genetic diseases are induced by mutations disturbing the maturation of pre-mRNAs, often affecting splicing. Antisense oligoribonucleotides (AONs) have been used to modulate splicing thereby circumventing the deleterious effects of mutations.
Agathe Eckenfelder +5 more
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Transport Oligonucleotides—A Novel System for Intracellular Delivery of Antisense Therapeutics
Molecules, 2020 Biological activity of antisense oligonucleotides (asON), especially those with a neutral backbone, is often attenuated by poor cellular accumulation. In the present proof-of-concept study, we propose a novel delivery system for asONs which implies the ...
Oleg V. Markov +9 more
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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
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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
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