Results 1 to 10 of about 1,703,096 (348)
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
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Machine Learning To Predict Cell-Penetrating Peptides for Antisense Delivery [PDF]
ACS Central Science, 2018 Cell-penetrating peptides (CPPs) can facilitate the intracellular delivery of large therapeutically relevant molecules, including proteins and oligonucleotides.
Justin M. Wolfe +6 more
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Cells, 2023 Antisense oligonucleotide-based (ASO) therapeutics have emerged as a promising strategy for the treatment of human disorders. Charge-neutral PMOs have promising biological and pharmacological properties for antisense applications.
Umme Sabrina Haque, Toshifumi Yokota
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The Challenges and Strategies of Antisense Oligonucleotide Drug Delivery
Biomedicines, 2021 Antisense oligonucleotides (ASOs) are used to selectively inhibit the translation of disease-associated genes via Ribonuclease H (RNaseH)-mediated cleavage or steric hindrance.
Maria Gagliardi, Ana Tari Ashizawa
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Delivery of Antisense Oligonucleotides to the Cornea
Nucleic Acid Therapeutics, 2020 Antisense oligonucleotides (ASOs) are synthetic nucleic acids that recognize complementary RNA sequences inside cells and modulate gene expression. In this study, we explore the feasibility of ASO delivery to the cornea.
Viet Q. Chau +8 more
semanticscholar +4 more sources
Antisense delivery using protamine-oligonucleotide particles. [PDF]
Nucleic Acids Research, 2000 Protamine, a polycationic peptide (mol. wt 4000-4500), was evaluated as a potential penetration enhancer for phosphodiester antisense oligonucleotides (ODNs). Unique complexes in the form of nanoparticles were spontaneously formed, which we call 'proticles'.
M. Junghans, J. Kreuter, A. Zimmer
semanticscholar +3 more sources
Delivery is key: lessons learnt from developing splice‐switching antisense therapies
EMBO Molecular Medicine, 2017 The use of splice‐switching antisense therapy is highly promising, with a wealth of pre‐clinical data and numerous clinical trials ongoing. Nevertheless, its potential to treat a variety of disorders has yet to be realized. The main obstacle impeding the
Caroline Godfrey +17 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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Imaging-Assisted Antisense Oligonucleotide Delivery for Tumor-Targeted Gene Therapy
Chemical & Biomedical ImagingAntisense oligonucleotide (ASO) represents a class of practical tools for targeting undruggable oncogenes with several candidates currently undergoing clinical investigation. The advancement of antisense therapeutics necessitates comprehensive approaches
Hanwen Liao +6 more
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Molecular Therapy: Nucleic Acids, 2020 The efficient delivery of antisense oligonucleotides (ASOs) to the targeted cells and organs remains a challenge, in particular, in vivo. Here, we investigated the ability of a library of biodegradable lipid nanoparticles (LNPs) in delivering ASO to both
Liu Yang +5 more
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