RNA Therapeutics
A special issue of Biomolecules (ISSN 2218-273X). This special issue belongs to the section "Biomacromolecules: Nucleic Acids".
Deadline for manuscript submissions: 30 June 2024 | Viewed by 5852
Special Issue Editor
Interests: antisense; drug delivery; antibiotics; peptide nucleic acid; gene targeting
Special Issues, Collections and Topics in MDPI journals
Special Issue Information
Dear Colleagues,
RNA therapeutics is one of the most rapidly expanding new drug technology platforms, showcasing very promising potential for a broad and increasing variety of indications. This is not least due to a simple generic, rational drug design for indications where a genetic cause has been established and characterized at the sequence level. RNA therapeutics is a broad field covering drugs based on nucleic acids and their analogues and derivatives targeting specific functional RNAs or DNA in the cell based on sequence-specific nucleic acid recognition, or by employing functional nucleic acids (e.g. mRNA of tRNA) as drugs themselves. Historically, the field dates back more than 30 years to when small synthetic antisense oligonucleotides were successfully developed to silence specific genes post transcriptionally at the mRNA level, and were then later used as specific modulators of pre-mRNA splicing. Approval for the first antisense drug (Mipomersen) was obtained by the FDA in 2013. Subsequently, following the discovery of small interfering RNAs (siRNAs/microRNAs) as post transcriptional gene regulators in eukaryotic cells, siRNAs were developed as gene-specific silencing drugs. The first siRNA drug was approved in 2018; overall, more than a dozen RNA therapeutics have within the last ten years been approved for human clinical use by the FDA, and many more are in the pre-clinical and clinical trial pipeline. Significant efforts are also being invested in developing RNA therapeutics for targeted gene editing, although no drugs have yet been approved.
This Special Issue will focus broadly on new discoveries and developments in RNA therapeutics, such as new targets, chemistries and mode of action principles, and not least on improving in vivo delivery, tissue targeting and the pharmacokinetic/dynamic properties of nucleic acid-based drugs. However, this issue will not cover vector-mediated gene therapy, CRISPR/Cas systems, aptamers or CpG oligonucleotides.
Prof. Dr. Peter E. Nielsen
Guest Editor
Manuscript Submission Information
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Submitted manuscripts should not have been published previously, nor be under consideration for publication elsewhere (except conference proceedings papers). All manuscripts are thoroughly refereed through a single-blind peer-review process. A guide for authors and other relevant information for submission of manuscripts is available on the Instructions for Authors page. Biomolecules is an international peer-reviewed open access monthly journal published by MDPI.
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Keywords
- antisense oligonucleotides (ASO)
- siRNA
- mRNA new chemistries
- therapeutic targets/indications
- cellular delivery/endosomal escape (LNPs/CPPs)
- in vivo administration/formulation/efficacy
- tissue targeting/PK/PD
