Delivery of Oligonucleotide-Based Therapeutics
A special issue of Pharmaceutics (ISSN 1999-4923). This special issue belongs to the section "Gene and Cell Therapy".
Deadline for manuscript submissions: 30 June 2024 | Viewed by 125
Special Issue Editors
Interests: drug delivery; gene delivery; nanomedicine; drug discovery; cancer research
Special Issue Information
Dear Colleagues,
Oligonucleotides are short nucleic acids that are capable of modulating gene expression via a range of different processes. This expands their range of targets beyond what is accessible to small molecule drugs. The different oligonucleotide-based therapeutic platforms include antisense oligonucleotides (ASOs), splice-correcting oligonucleotides (SCOs) and RNA interference (RNAi) including small interfering RNA (siRNA), short hairpin RNA (shRNA), and microRNA (miRNA).
The FDA approval of the first RNAi-based drug, Patisiran, in 2018, started a new era in oligonucleotide-based therapies. As of today, ten oligonucleotide-based drugs have received FDA approval and many more are under investigation for treatment of various diseases ranging from cancer and rare metabolic disease to cardiovascular and neurodegenerative diseases. However, the current delivery strategies mainly result in high abundance of oligonucleotides in the liver with low distribution to other tissues, which is a major obstacle for widespread clinical translation of oligonucleotide-based therapeutics. Therefore, development of advanced delivery platforms for highly efficient and targeted delivery of oligonucleotides to the extrahepatic tissues is one of the key challenges in gene therapy.
The aim of this Special Issue of Pharmaceutics is to present research papers addressing novel strategies for highly efficient delivery of oligonucleotide‐based therapeutics to different organs and tissues.
Dr. Roja Hadianamrei
Dr. David Rusling
Guest Editors
Manuscript Submission Information
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Keywords
- oligonucleotide
- RNA interference
- small interfering RNA
- short hairpin RNA
- splice-correcting oligonucleotide
- gene delivery
