Results 31 to 40 of about 47,774 (288)

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
doaj   +1 more source

Polyquaternium-mediated delivery of morpholino oligonucleotides for exon-skipping in vitro and in mdx mice

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, Bo Wu, Sapana N Shah, Peijuan Lu, Qilong Lu   +4 more
doaj   +1 more source

Enhancing Antisense Oligonucleotide-Based Therapeutic Delivery with DG9, a Versatile Cell-Penetrating Peptide

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   +1 more source

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, Lourdes R Desviat, Bård Smedsrød, France Piétri‐Rouxel, Michela A Denti, Petra Disterer, Stéphanie Lorain, Gisela Nogales‐Gadea, Valentina Sardone, Rayan Anwar, Samir EL Andaloussi, Taavi Lehto, Bernard Khoo, Camilla Brolin, Willeke MC van Roon‐Mom, Aurélie Goyenvalle, Annemieke Aartsma‐Rus, Virginia Arechavala‐Gomeza   +17 more
doaj   +1 more source

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, Craig S. McIntosh, Kristin A. West, Sue Fletcher, Steve D. Wilton   +4 more
doaj   +1 more source

Imaging-Assisted Antisense Oligonucleotide Delivery for Tumor-Targeted Gene Therapy

Chemical & Biomedical Imaging
Antisense 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, Siyi Wang, Xiaoning Wang, David Zixiang Dai, Yan Zhang, Chenghong Zhu, Jinbo Li   +6 more
doaj   +2 more sources

An Investigation into the Potential of Targeting Escherichia coli rne mRNA with Locked Nucleic Acid (LNA) Gapmers as an Antibacterial Strategy

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, Charlotte E. Mardle, Hassan Gneid, Ciara G. Ball, Darren M. Gowers, Helen S. Atkins, Louise E. Butt, Jonathan K. Watts, Helen A. Vincent, Anastasia J. Callaghan   +9 more
doaj   +1 more source

The Cellular Processing Capacity Limits the Amounts of Chimeric U7 snRNA Available for Antisense Delivery

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, Julie Tordo, Arran Babbs, Kay E Davies, Aurélie Goyenvalle, Olivier Danos   +5 more
doaj   +1 more source

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, Anton V. Filatov, Maxim S. Kupryushkin, Ivan V. Chernikov, Olga A. Patutina, Anton A. Strunov, Elena L. Chernolovskaya, Valentin V. Vlassov, Dmitrii V. Pyshnyi, Marina A. Zenkova   +9 more
doaj   +1 more source

Meeting review : ESF workshop on "Impact of nucleic acid chemistry on gene function analysis: antisense, aptamers, ribozymes and RNAi"

, 2008
The shortage of functional information compared to the abundance of sequence information characterizes today’s situation in functional genomics. For many years the knock-down of a gene’s product has been the most powerful way of analysing its function ...
Prydz, Hans, Reinhard-Rupp, Jutta, Heermeier, Kathrin, Engels, Joachim W. (Prof. Dr.)   +3 more
core   +1 more source