Strategies for Expanding the Therapeutic Potential of Oligonucleotides
A special issue of Biomedicines (ISSN 2227-9059). This special issue belongs to the section "Molecular and Translational Medicine".
Deadline for manuscript submissions: closed (30 June 2019) | Viewed by 143
Special Issue Editor
Interests: therapeutic development; chemical modification; and targeted delivery systems for oligonucleotides; circulating nucleic acid biomarkers; bioanalysis; cardiovascular diseases
Special Issue Information
Dear Colleagues,
Recent successful marketing applications of therapeutic oligonucleotides (nusinersen, patisiran, inotersen) show that the technology has now reached clinical relevance. Issues such as poor pharmacokinetic properties have largely been overcome by the careful optimization of chemical modification patterns, target and sequence selection, and delivery systems. Nevertheless, the therapeutic benefit is still restricted, particularly to local administration to the site of action or to silencing hepatic targets after systemic application. Therefore, further progress is required, including on the chemico-physical properties, the definition of useful disease targets that are amenable for oligonucleotides, and improved delivery. More efficient organ- and tissue-targeted delivery for non-liver targets would constitute a major improvement. The modulation of non-coding RNA targets and the successful application of mRNA therapies may further expand the potential of synthetic oligonucleotide compounds.
This Special Issue aims to include original research and reviews covering all aspects of the therapeutic development of oligonucleotides, with special attention paid to the translational potential. Studies focusing on new delivery systems and oligonucleotide bioconjugates, targeted delivery, off-target effects, as well as the identification of new potential disease-relevant gene and ncRNA targets and mechanisms are welcome.
Dr. Johannes Winkler
Guest Editor
Manuscript Submission Information
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Keywords
- antisense
- biomolecular targeting
- miRNA, non-coding RNA
- off-target effects
- oligonucleotide delivery
- RNA interference
