The Challenges and Strategies of Antisense Oligonucleotide Drug Delivery [PDF]
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 and uptake of free and liposome-encapsulated cholesterol-conjugated antisense oligonucleotides in Atlantic salmon sperm: insights from high-resolution imaging [PDF]
BMC Veterinary ResearchThe development of efficient methods for large-scale delivery of antisense molecules to Atlantic salmon (Salmo salar L.) eggs remains a significant challenge, limiting the application of antisense technologies in salmon aquaculture at an industrial scale.
Jaya Kumari Swain +6 more
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Graphene Oxide–Antisense miR-21 Nanosystem Modulates Gene Expression and Suppresses Tumorigenesis in HepG2-Derived CAM Xenografts [PDF]
BiomoleculesGraphene oxide (GO) is a promising nanocarrier for the delivery of oligonucleotides. It offers a high loading capacity, efficient cellular uptake, and surface functionalization.
Paola Trischitta +5 more
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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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Two problems in antisense biotechnology: in vitro delivery and the design of antisense experiments
Biochimica Et Biophysica Acta Gene Regulatory Mechanisms, 1999 Antisense oligonucleotides are invaluable reagents for the specific downregulation of gene expression. In the absence of a carrier, charged oligonucleotides (e.g., phosphorothioates) can interact with a large number of cell surface proteins, but tend to be internalized into the endosomal/lysosomal compartment.
C A Stein, C A Stein
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Simultaneous Down-Regulation of Intracellular hTERT and GPX4 mRNA Using MnO2-Nanosheet Probes to Induce Cancer Cell Death [PDF]
SensorsCancer remains a leading global cause of death, with conventional treatments often limited by toxicity and recurrence. Recent advances in gene therapy and nanodrug delivery offer new avenues for precision oncology. Human telomerase reverse transcriptase (
Yixin Miao +3 more
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Machine Learning To Predict Cell-Penetrating Peptides for Antisense Delivery [PDF]
ACS Central Science, 2018 Justin M. Wolfe +6 more
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Targeted SMN Exon Skipping: A Useful Control to Assess In Vitro and In Vivo Splice-Switching Studies
Biomedicines, 2021 The literature surrounding the use of antisense oligonucleotides continues to grow, with new disease and mechanistic applications constantly evolving. Furthermore, the discovery and advancement of novel chemistries continues to improve antisense delivery,
Loren L. Flynn +6 more
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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. We used quantitative polymerase chain reaction to test the efficacy of a benchmark ASO targeting a noncoding ...
Viet Q, Chau +8 more
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Challenges and future perspective of antisense therapy for spinal muscular atrophy: A review
European Journal of Cell Biology, 2023 Spinal muscular atrophy (SMA), the most common genetic cause of infantile death, is caused by a mutation in the survival of motor neuron 1 gene (SMN1), leading to the death of motor neurons and progressive muscle weakness.
Zorica Nakevska, Toshifumi Yokota
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