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
doaj +5 more sources
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
doaj +2 more sources
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
doaj +2 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
doaj +2 more sources
RNA Therapeutics Targeting Skeletal Muscle: Emerging Antisense and Gene-Modifying Strategies [PDF]
BiomoleculesRNA-based therapeutics are reshaping the treatment landscape for skeletal muscle disorders by enabling modulation of RNA processing or direct correction of disease-causing alleles.
Takayuki Kuroda, Toshifumi Yokota
doaj +2 more sources
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 +3 more sources
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
doaj +3 more sources
Scavenger receptor–mediated lung-targeted delivery of anti-miR-155 oligoDNA nanomicelles with curcumin for acute lung injury therapy [PDF]
Asian Journal of Pharmaceutical SciencesAntisense oligonucleotides offer a powerful strategy for suppressing pro-inflammatory microRNAs, but efficient long-term delivery after systemic administration remains challenging.
Minji Kang +3 more
doaj +2 more sources
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
doaj +1 more source
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
openaire +3 more sources